Clinical Trials - Gene Therapy Trial Browser
The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, meeting abstracts, and more to give a comprehensive overview of the gene therapy clinical trial landscape.
Showing 1-88 of 88 results in ClinicalTrials
SCGE Platform Gene Therapy Clinical Trials downloaded on: 2026/08/30 16:45:11; Please cite the Somatic Cell Genome Editing Consortium Platform when using publicly accessible data in formal presentation or publication.
Definitions
Abbreviation
| AAV | Adeno-associated virus, e.g. AAV2, AAV5, AAV2/5 indicates virus containing the genome of serotype 2 packaged in the capsid from serotype 5 |
| ABE | Adenine base editor |
| Ad | Adenovirus |
| Cas9 | CRISPR associated protein 9 |
| CBE | Cytosine base editor |
| CRISPR | Clustered regularly interspaced short palindromic repeats |
| Gc | Genome copies |
| HIV | Human immunodeficiency virus |
| HSV | Herpes simplex virus |
| LNP | Lipid nanoparticle |
| LV | Lentivirus |
| MRNA | Messenger ribonucleic acid |
| ODD | Orphan Drug Designation |
| PFU | Plaque forming units |
| RMAT | Regenerative Medicine Advanced Therapy |
| RNP | Ribonucleoprotein |
| RPDD | Rare Pediatric Disease Designation |
| RV | Retrovirus |
| START | Support for Clinical Trials Advancing Rare Disease Therapeutics |
| Vg | Vector genomes |
| VSV-G | Vesicular stomatitis virus G |
Delivery Type
| Electroporation | Cell membrane permeablized by electrical field to allow gene therapy to enter the cell |
| Lipid encapsulation | Any lipid nanoparticle used to deliver editor, corrected gene |
| Microinjection | Drug is injected into individual cells |
| Plasmid | Any plasmid used to deliver editor, corrected gene |
| Viral transduction | Any virus used to deliver editor, corrected gene, etc. |
FDA Designation
| Accelerated Approval | These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint |
| Breakthrough Therapy | A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy. |
| Fast Track | Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need |
| Orphan Drug | This designation is for drugs intended to treat rare diseases or conditions that affect a small number of people, offering incentives like tax credits and market exclusivity |
| Priority Review | A Priority Review designation means FDA’s goal is to take action on an application within 6 months. |
| Rare Pediatric Disease | The rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases. |
| Regenerative Medicine Advanced Therapy | Facilitates the development and expedites the review of regenerative medicine therapies, including cell therapies, therapeutic tissue engineering products, and human cell and tissue products, for serious or life-threatening conditions. |
| Support for Clinical Trials Advancing Rare Disease Therapeutics | (START) Pilot Program is a program designed to accelerate the development of novel drug and biological products for rare diseases by providing sponsors with enhanced communication and guidance from FDA staff. |
Funder Type
| Industry | All for-profit entities |
| NIH | U.S. National Institutes of Health |
| Other Non-Profit | Includes individuals, universities, community-based organizations |
Route Of Administration
| CED | Convection enhanced delivery to the brain |
| Inhalational | Delivered to the lungs in the form of a fine spray |
| Intraarterial | Into the lumen of an artery |
| Intraarticular | Into a joint space |
| Intracerebroventricular | Into the ventricles of the brain (ICV) |
| Intracisterna magna | Into the cisterna magna of the brain |
| Intracochlear | Into the cochlea of the ear |
| Intradermal | Into the dermal layer of the skin |
| Intragastric | Into the stomach |
| Intramuscular | Into a skeletal muscle |
| Intraocular | Administered into the eye |
| Intraparenchymal | Into the brain |
| Intraperitoneal | Into the peritoneal cavity |
| Intrastromal | Into the stroma of the cornea |
| Intrathecal | Into the spinal canal |
| Intravenous | Into a vein |
| Intravesicular | Into the bladder |
| Intravitreal | Into the eye (IVT) |
| Subcutaneous | Under the skin |
| Subretinal | Under the sensory retina |
| Suprachoroidal | Into the suprachoroidal space between the sclera and the choroid of the eye |
| Topical | Applied to the outer layer of the skin |
Stages
| Early Phase I | Describes exploratory trials conducted before traditional Phase I trials to investigate how or whether a drug affects the body, have no therapeutic or diagnostic goals |
| Phase I | Describes clinical trials that focus on the safety of a drug |
| Phase II | Describes clinical trials that gather preliminary data on effectiveness, continue to monitor safety |
| Phase I/II | Combination Phase I/Phase II clinical trial |
| Phase III | Pivotal experiments to gather data on safety and effectiveness |
| Phase II/III | Combination Phase II/Phase III clinical trial |
Study Status
| Active, Not Recruiting | Study has ongoing, but is not enrolling new participants |
| Completed | Study has concluded normally |
| Not Yet Recruiting | Study has not started enrollment |
| Recruiting | Study is actively looking for participants |
| Suspended | Study halted prematurely but has the potential to resume |
| Terminated | Study was halted prematurely and will not resume, participants are no longer recieving intervention |
| Unknown | Study has passed its completion date, but last known status was not listed as Completed, Terminated or Withdrawn. Status has not been verified within the past 2 years. Studies with an unknown status are considered closed studies |
Table Column Header
| Actual Study Start Date (m/d/y) | The actual date on which the first participant was enrolled in a clinical study |
| Adult/Pediatric/Both | Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both |
| Ages Eligible for Study | More specific age ranges eligible for the study |
| Clinical Centers in USA? | Binary variable on whether trial sites are located in the USA (Y) or not (N) |
| Clinical Publications | URL connections to clinical data on human subjects |
| Compound Name | The interventional compound given to the study subjects |
| Countries | List of countries that contain at least 1 clinical trial site |
| Current Stage | The stage of the clinical trial, determined based on the studies' objective |
| Date of Last Update | The most recent date on which changes to a study record were made available on ClinicalTrials.gov |
| Delivery System | Describes the nature of the drug substance or process that is used to deliver the editor or corrected gene |
| Development Status | Indicates whether or not the clinical development program is ongoing, or if the drug is approved |
| Dose levels (up to 5) | List of doses given in the indicated clinical trial, may be expressed in specific units, or a range of possible doses |
| Drug Product Type | Describes the nature of the drug product |
| Editor Type | For gene editing type therapies, the protein that will perform the gene correction |
| Estimated Primary Completion Date (m/d/y) | The anticipated date that the primary outcome measure data will be complete (last participant data collected) |
| FDA Designations | List of special FDA designations granted to the Sponsor for the development program (i.e. Orphan Drug Designation, Fast Track, Rare Pediatric Disease Designation, RMAT, etc.) |
| Funder Type | Describes the organization that provides support for the clinical study |
| Grants | URL connections to funding used to conduct preclinical or clinical studies, granted by NIH or other US institution |
| Has US IND? | Binary variable indicating whether the drug product is regulated by an approved Investigational New Drug application by the Food and Drug Administration of the United States |
| Indication | The disease, disorder, syndrome, illness, or injury that is being studied |
| Locations | List of countries that contain at least 1 clinical trial site |
| Mechanism of Action | Simplified description of how the drug product works |
| NCT Number | Unique identification code given to each clinical study upon registration at ClinicalTrials.gov |
| News and Press Releases | URL connections to press releases generated by drug product Sponsor |
| N trial sites | Number of clinical sites where the clinical trial is conducted |
| Patents | URL connections to patents, intellectual property related to the drug product |
| Phases | The stage of the clinical trial, determined based on the studies' objective |
| Preclinical Publications | URL connections to preclinical data (in vitro, animal data) |
| Protocols | URL connections to clinical trial protocols, study design papers |
| Recent Regulatory Updates | News, updates on recent or anticipated regulatory milestones |
| Recruitment Status | Indicates the current recruitment status or the expanded access status |
| Results Posted | Indicates if summary results are posted to the clinical trial record |
| Route of Administration | How the drug product is introduced to the body |
| Sexes Eligible for Study | A type of eligibility criteria that indicates the sex of people who may participate in a clinical study (all, female, male) |
| Sponsor | The organization or person who initiates the study and who has authority and control over the study |
| Sponsor Class | Describes the organization that provides support for the clinical study |
| Standard Ages | Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both |
| Target Gene or Variant | The symbol of the gene that is corrected or replaced by the drug compound |
| Target Tissue or Cell | Lists target cells if the therapy is directed at a particular cell type (either by ex-vivo enrichment, or tissue-specific regulatory elements) |
| Therapy Route | Describes whether the gene therapy is introduced to cells in-vivo or ex-vivo |
| Therapy Type | Describes the nature of the gene therapy |
| Trial Enrollment | Number of study subjects planned or actually enrolled in the study |
| Vector Type | Gives additional specifics (if known) about delivery system (e.g. AAV serotype) |
Therapy Route
| Ex-vivo | When the cells are modified outside the body |
| In-vivo | When the cells are modified inside the body |
Therapy Type
| Gene editing | A gene therapy where disease-causing variant is corrected via a gene editor |
| Recent Regulatory Updates | A gene therapy where a corrected gene is administered to relevant cells/tissues via a delivery system |
References
Some text in the modal.
Glycogen Storage Disease Type Ia
Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Compound
GENGLYCOS
Therapeutic Modality
Gene transfer
Target Gene
G6PC
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-07-14
Last Modified Date: 1787634000000
Enrollment: 49
Brazil, Canada, Denmark + 6 more
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor
Janssen Research & Development, LLC (Industry)
Compound
Botaretigene sparoparvovec
Therapeutic Modality
Gene transfer
Target Gene
RPGR
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2020-11-05
Last Modified Date: 1787115600000
Enrollment: 105
Belgium, Canada, Denmark + 8 more
Fabry Disease
Sponsor
Sangamo Therapeutics (Industry)
Compound
ST-920
Therapeutic Modality
Gene transfer
Target Gene
GLA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2019-08-01
Last Modified Date: 1786683600000
Enrollment: 36
Australia, Canada, Germany + 4 more
Parkinson's Disease
Sponsor
MeiraGTx, LLC (Industry)
Compound
AAV-GAD
Therapeutic Modality
Gene transfer
Target Gene
GAD1/2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2022-10-12
Last Modified Date: 1786424400000
Enrollment: 14
United States
Alzheimer's Disease, Early Onset Alzheimer's Disease
Sponsor
Lexeo Therapeutics (Industry)
Compound
LX1001
Therapeutic Modality
Gene transfer
Target Gene
APOE2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track
First Submitted: 2018-08-03
Last Modified Date: 1786424400000
Enrollment: 15
United States
Amyotrophic Lateral Sclerosis (ALS)
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2021-11-05
Last Modified Date: 1784869200000
Enrollment: 8
South Korea, United States
Charcot-Marie-Tooth Disease 1A
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2022-03-31
Last Modified Date: 1784869200000
Enrollment: 12
South Korea
Peripheral Artery Disease
Sponsor
Northwestern University (Other)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2017-11-30
Last Modified Date: 1784869200000
Enrollment: 39
United States
Critical Limb Ischemia
Sponsor
Beijing Northland Biotech. Co., Ltd. (Industry)
Compound
NL003
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2020-01-18
Last Modified Date: 1784869200000
Enrollment: 302
China
Ischemic Heart Disease
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2011-08-21
Last Modified Date: 1784869200000
Enrollment: 9
South Korea
Critical Limb Ischemia
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2010-02-04
Last Modified Date: 1784869200000
Enrollment: 52
South Korea, United States
Osteoarthritis, Knee
Sponsor
Xalud Therapeutics, Inc. (Industry)
Compound
XT-150
Therapeutic Modality
Gene transfer
Target Gene
Recombinant IL10
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track
First Submitted: 2019-10-09
Last Modified Date: 1784869200000
Enrollment: 289
Australia, United States
Diabetic Neuropathy
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2020-06-25
Last Modified Date: 1784869200000
Enrollment: 162
United States
Neovascular (Wet) Age-Related Macular Degeneration
Sponsor
Oxford BioMedica (Industry)
Compound
RetinoStat
Therapeutic Modality
Gene transfer
Target Gene
COL18A1+PLG
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2011-02-21
Last Modified Date: 1784782800000
Enrollment: 21
United States
Choroideremia
Sponsor
Spark Therapeutics, Inc. (Industry)
Compound
SPK-7001
Therapeutic Modality
Gene transfer
Target Gene
CHM
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2015-01-12
Last Modified Date: 1784782800000
Enrollment: 15
United States
HIV-1-infection
Sponsor
Excision BioTherapeutics (Industry)
Compound
EBT-101
Therapeutic Modality
Gene editing
Target Gene
HIV genes
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Gene excision
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2021-11-22
Last Modified Date: 1784782800000
Enrollment: 6
United States
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor
Adverum Biotechnologies, Inc. (Industry)
Compound
ADVM-043
Therapeutic Modality
Gene transfer
Target Gene
SERPINA1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-06-10
Last Modified Date: 1784782800000
Enrollment: 6
United States
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Genzyme, a Sanofi Company (Industry)
Compound
RAAV2-sFLT01
Therapeutic Modality
Gene transfer
Target Gene
SFLT01
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2009-12-02
Last Modified Date: 1784696400000
Enrollment: 19
United States
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor
Biogen (Industry)
Compound
BIIB112
Therapeutic Modality
Gene transfer
Target Gene
RPGR
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2017-03-29
Last Modified Date: 1784696400000
Enrollment: 50
United Kingdom, United States
Idiopathic Parkinson's Disease
Sponsor
Sangamo Therapeutics (Industry)
Compound
CERE-120
Therapeutic Modality
Gene transfer
Target Gene
NRTN
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2009-09-25
Last Modified Date: 1784696400000
Enrollment: 57
United States
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor
University of Florida (Other)
Compound
AAV1-CMV-hGAA
Therapeutic Modality
Gene transfer
Target Gene
GAA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-07-13
Last Modified Date: 1784696400000
Enrollment: 9
United States
Spinal Muscular Atrophy
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
ITVISMA
Therapeutic Modality
Gene transfer
Target Gene
SMN1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Orphan Drug Designation
First Submitted: 2022-05-18
Last Modified Date: 1784696400000
Enrollment: 27
Australia, Belgium, Canada + 6 more
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)
Sponsor
Emily de los Reyes (Other)
Compound
AT-GTX-501
Therapeutic Modality
Gene transfer
Target Gene
CLN6
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-16
Last Modified Date: 1784696400000
Enrollment: 13
United States
Hemophilia B
Sponsor
CSL Behring (Industry)
Compound
HEMGENIX
Therapeutic Modality
Gene transfer
Target Gene
F9-R338L
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Priority Review, Orphan Drug Designation
First Submitted: 2018-06-14
Last Modified Date: 1784696400000
Enrollment: 67
Belgium, Denmark, Germany + 5 more
Alzheimer's Disease
Sponsor
Sangamo Therapeutics (Industry)
Compound
CERE-110
Therapeutic Modality
Gene transfer
Target Gene
NGF
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2009-04-03
Last Modified Date: 1784696400000
Enrollment: 49
United States
Diabetic Macular Edema, Diabetic Retinopathy
Sponsor
Adverum Biotechnologies, Inc. (Industry)
Compound
Ixoberogene soroparvovec
Therapeutic Modality
Gene transfer
Target Gene
Codon optimized aflibercept
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2020-05-28
Last Modified Date: 1784696400000
Enrollment: 36
Puerto Rico, United States
Hemophilia A
Sponsor
BioMarin Pharmaceutical (Industry)
Compound
ROCTAVIAN
Therapeutic Modality
Gene transfer
Target Gene
F8
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-03-24
Last Modified Date: 1784696400000
Enrollment: 22
Australia, Brazil, Taiwan + 1 more
Leber Congenital Amaurosis
Sponsor
Nantes University Hospital (Other)
Compound
RAAV2/4.hRPE65
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-11-24
Last Modified Date: 1784696400000
Enrollment: 9
France
Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration
Sponsor
Nanoscope Therapeutics Inc. (Industry)
Compound
MCO-010
Therapeutic Modality
Gene transfer
Target Gene
MCO
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2021-06-15
Last Modified Date: 1784696400000
Enrollment: 27
Puerto Rico, United States
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Exegenesis Bio (Industry)
Compound
EXG102-031
Therapeutic Modality
Gene transfer
Target Gene
ABD-VEGFR
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2023-06-06
Last Modified Date: 1784696400000
Enrollment: 12
United States
X-Linked Retinoschisis
Sponsor
Beacon Therapeutics (Industry)
Compound
RAAV2tYF-CB-hRS1
Therapeutic Modality
Gene transfer
Target Gene
RS1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2015-04-02
Last Modified Date: 1784696400000
Enrollment: 27
United States
Achromatopsia
Sponsor
MeiraGTx UK II Ltd (Industry)
Compound
AAV-CNGA3
Therapeutic Modality
Gene transfer
Target Gene
CNGA3
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-11-27
Last Modified Date: 1784696400000
Enrollment: 11
United Kingdom, United States
Sickle Cell Disease
Sponsor
Donald B. Kohn, M.D. (Other)
Compound
LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Therapeutic Modality
Gene transfer
Target Gene
HBB
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2014-09-20
Last Modified Date: 1784696400000
Enrollment: 4
United States
Advanced Retinitis Pigmentosa
Sponsor
AbbVie (Industry)
Compound
RST-001
Therapeutic Modality
Gene transfer
Target Gene
ChR2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Orphan Drug Designation
First Submitted: 2015-09-21
Last Modified Date: 1784696400000
Enrollment: 14
United States
Leber Congenital Amaurosis
Sponsor
MeiraGTx UK II Ltd (Industry)
Compound
Cevaretigene ritoparvovec
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-04-28
Last Modified Date: 1784696400000
Enrollment: 15
United Kingdom, United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Megan Waldrop (Other)
Compound
AT702
Therapeutic Modality
Gene transfer
Target Gene
DMD (exon 2)
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Exon skipping/splice editor
First Submitted: 2020-01-22
Last Modified Date: 1784696400000
Enrollment: 3
United States
Stargardt Disease
Sponsor
Nanoscope Therapeutics Inc. (Industry)
Compound
MCO-010
Therapeutic Modality
Gene transfer
Target Gene
MCO
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2022-06-09
Last Modified Date: 1784696400000
Enrollment: 6
United States
Sickle Cell Disease
Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Compound
DREPAGLOBE
Therapeutic Modality
Gene transfer
Target Gene
HBB
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2019-05-14
Last Modified Date: 1784696400000
Enrollment: 6
France
Spinal Muscular Atrophy
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
ZOLGENSMA
Therapeutic Modality
Gene transfer
Target Gene
SMN1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-09-20
Last Modified Date: 1784696400000
Enrollment: 16
Argentina, Brazil
X-Linked Retinoschisis
Sponsor
VegaVect, Inc. (Industry)
Compound
RS1 AAV Vector
Therapeutic Modality
Gene transfer
Target Gene
RS1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-12-16
Last Modified Date: 1784696400000
Enrollment: 12
United States
Leukocyte Adhesion Defect - Type I
Sponsor
Rocket Pharmaceuticals Inc. (Industry)
Compound
KRESLADI
Therapeutic Modality
Gene transfer
Target Gene
ITGB2
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-01-18
Last Modified Date: 1784696400000
Enrollment: 9
Spain, United Kingdom, United States
Cerebral Adrenoleukodystrophy (CALD)
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
SKYSONA
Therapeutic Modality
Gene transfer
Target Gene
ABCD1
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-02-13
Last Modified Date: 1784696400000
Enrollment: 35
France, Germany, Italy + 3 more
Metachromatic Leukodystrophy
Sponsor
Institut National de la Santé Et de la Recherche Médicale, France (Other gov)
Compound
AAVrh.10-CAG/cuARSA
Therapeutic Modality
Gene transfer
Target Gene
ARSA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2013-01-28
Last Modified Date: 1784696400000
Enrollment: 5
France
Acute Intermittent Porphyria
Sponsor
Digna Biotech S.L. (Industry)
Compound
RAAV2/5-PBGD
Therapeutic Modality
Gene transfer
Target Gene
PBGD
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-02-28
Last Modified Date: 1784696400000
Enrollment: 8
Spain
Hemophilia B
Sponsor
St. Jude Children's Research Hospital (Other)
Compound
ScAAV2/8-LP1-hFIXco
Therapeutic Modality
Gene transfer
Target Gene
F9
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-09-16
Last Modified Date: 1784696400000
Enrollment: 14
United Kingdom, United States
Geographic Atrophy
Sponsor
Janssen Research & Development, LLC (Industry)
Compound
JNJ-1887
Therapeutic Modality
Gene transfer
Target Gene
CD59
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2023-03-07
Last Modified Date: 1784696400000
Enrollment: 305
Australia, Belgium, Canada + 14 more
Leber Congenital Amaurosis-Type 2
Sponsor
University College, London (Other)
Compound
TgAAG76
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2008-03-20
Last Modified Date: 1784696400000
Enrollment: 12
United Kingdom
Leber Congenital Amaurosis-Type 2
Sponsor
Hadassah Medical Organization (Other)
Compound
RAAV2-hRPE65
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-01-12
Last Modified Date: 1784696400000
Enrollment: 3
Israel
Leber Hereditary Optic Neuropathy (LHON)
Sponsor
GenSight Biologics (Industry)
Compound
LUMEVOQ
Therapeutic Modality
Gene transfer
Target Gene
ND4 m.11778G>A
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2016-01-07
Last Modified Date: 1784696400000
Enrollment: 39
France, Germany, Italy + 2 more
Duchenne Muscular Dystrophy (DMD)
Sponsor
Kevin Flanigan (Other)
Compound
RAAVrh74.MCK.GALGT2
Therapeutic Modality
Gene transfer
Target Gene
GALGT2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2017-11-01
Last Modified Date: 1784696400000
Enrollment: 2
United States
CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Sponsor
Weill Medical College of Cornell University (Other)
Compound
LX1004
Therapeutic Modality
Gene transfer
Target Gene
TPP1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2010-03-22
Last Modified Date: 1784696400000
Enrollment: 12
United States
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor
University of Florida (Other)
Compound
RAAV9-DES-hGAA
Therapeutic Modality
Gene transfer
Target Gene
GAA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-09-11
Last Modified Date: 1784696400000
Enrollment: 2
United States
Retinal Disease, Retinitis Pigmentosa
Sponsor
King Khaled Eye Specialist Hospital (Other gov)
Compound
AAV2-VMD2-hMERTK
Therapeutic Modality
Gene transfer
Target Gene
MERTK
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-09-28
Last Modified Date: 1784696400000
Enrollment: 6
Saudi Arabia
Leber Congenital Amaurosis-Type 2
Sponsor
Beacon Therapeutics (Industry)
Compound
RAAV2-CB-hRPE65
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2008-09-08
Last Modified Date: 1784696400000
Enrollment: 12
United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Nationwide Children's Hospital (Other)
Compound
RAAV2.5-CMV-mini-Dystrophin
Therapeutic Modality
Gene transfer
Target Gene
Micro-dystrophin
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2007-01-26
Last Modified Date: 1784696400000
Enrollment: 6
United States
Sanfilippo Syndrome B
Sponsor
UniQure Biopharma B.V. (Industry)
Compound
AMT-110
Therapeutic Modality
Gene transfer
Target Gene
NAGLU
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2016-06-15
Last Modified Date: 1784696400000
Enrollment: 4
France
Pompe Disease
Sponsor
AskBio Inc (Industry)
Compound
ACTUS-101
Therapeutic Modality
Gene transfer
Target Gene
GAA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track
First Submitted: 2018-04-13
Last Modified Date: 1784696400000
Enrollment: 7
United States
Mucopolysaccharidosis Type VI
Sponsor
Fondazione Telethon (Other)
Compound
AAV2/8.TBG.hARSB
Therapeutic Modality
Gene transfer
Target Gene
ARSB
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2017-04-12
Last Modified Date: 1784696400000
Enrollment: 9
Italy, Turkey (Türkiye)
X-Linked Retinoschisis
Sponsor
Zhongmou Therapeutics (Industry)
Compound
ZM-01
Therapeutic Modality
Gene transfer
Target Gene
RS1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2023-09-25
Last Modified Date: 1784696400000
Enrollment: 2
China
Parkinson's Disease
Sponsor
Neurocrine Biosciences (Industry)
Compound
VY-AADC01
Therapeutic Modality
Gene transfer
Target Gene
DDC
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-05-16
Last Modified Date: 1784696400000
Enrollment: 14
United States
Retinitis Pigmentosa
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
CPK850
Therapeutic Modality
Gene transfer
Target Gene
RLBP1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2017-12-11
Last Modified Date: 1784696400000
Enrollment: 12
Sweden
Beta-Thalassemia Major
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
ZYNTEGLO
Therapeutic Modality
Gene transfer
Target Gene
HBB
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-29
Last Modified Date: 1784696400000
Enrollment: 19
France, Germany, Greece + 3 more
Leber Hereditary Optic Neuropathy (LHON)
Sponsor
Byron Lam (Other)
Compound
ScAAV2-P1ND4v2
Therapeutic Modality
Gene transfer
Target Gene
ND4
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-06-06
Last Modified Date: 1784696400000
Enrollment: 28
United States
Choroideremia
Sponsor
Biogen (Industry)
Compound
BIIB111
Therapeutic Modality
Gene transfer
Target Gene
CHM
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-03-07
Last Modified Date: 1784696400000
Enrollment: 169
Canada, Denmark, Finland + 5 more
Unilateral OR Bilateral Severe to Profound Hearing Loss
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
CGF166
Therapeutic Modality
Gene transfer
Target Gene
ATOH1
Vector Type
Adenovirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2014-05-05
Last Modified Date: 1784696400000
Enrollment: 22
United States
Achromatopsia
Sponsor
MeiraGTx UK II Ltd (Industry)
Compound
AAV-CNGB3
Therapeutic Modality
Gene transfer
Target Gene
CNGB3
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-11-22
Last Modified Date: 1784696400000
Enrollment: 23
United Kingdom, United States
Hemophilia A
Sponsor
Christian Medical College, Vellore, India (Other)
Compound
CD68-ET3-LV
Therapeutic Modality
Gene transfer
Target Gene
F8
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2022-02-16
Last Modified Date: 1784696400000
Enrollment: 6
India
Cystic Fibrosis
Sponsor
Imperial College London (Other)
Compound
PGM169/GL67A
Therapeutic Modality
Gene transfer
Target Gene
CFTR
Delivery System
Endocytosis
Mechanism of Action
Functional gene replacement
First Submitted: 2012-06-14
Last Modified Date: 1784610000000
Enrollment: 130
United Kingdom
Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor
Genethon (Other)
Compound
AAV1.DES.hSGCG
Therapeutic Modality
Gene transfer
Target Gene
SGCG
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-03-17
Last Modified Date: 1784610000000
Enrollment: 9
France
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Compound
Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre
Therapeutic Modality
Gene transfer
Target Gene
IL2RG
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-06-21
Last Modified Date: 1784610000000
Enrollment: 5
France
Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies
Sponsor
Celladon Corporation (Industry)
Compound
AAV1-CMV-Serca2a
Therapeutic Modality
Gene transfer
Target Gene
SERCA2A
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2012-07-16
Last Modified Date: 1784610000000
Enrollment: 250
Belgium, Denmark, Germany + 7 more
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Sponsor
Nationwide Children's Hospital (Other)
Compound
RAAV1.CMV.huFollistatin344
Therapeutic Modality
Gene transfer
Target Gene
FST (FS344)
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2012-01-23
Last Modified Date: 1784610000000
Enrollment: 15
United States
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor
Krystal Biotech, Inc. (Industry)
Compound
VYJUVEK
Therapeutic Modality
Gene transfer
Target Gene
COL7A1
Vector Type
Herpesvirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-06-02
Last Modified Date: 1784610000000
Enrollment: 47
United States
Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Sponsor
Verve Therapeutics, Inc. (Industry)
Compound
VERVE-101
Therapeutic Modality
Gene editing
Target Gene
PCSK9
Delivery System
Endocytosis
Mechanism of Action
Gene inactivation
First Submitted: 2022-05-19
Last Modified Date: 1784610000000
Enrollment: 13
New Zealand, United Kingdom
Fanconi Anemia Complementation Group A
Sponsor
Boston Children's Hospital (Other)
Compound
MSCV-FANCA
Therapeutic Modality
Gene transfer
Target Gene
FANCA
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2006-01-04
Last Modified Date: 1784610000000
Enrollment: 3
United States
Congestive Heart Failure
Sponsor
Hammond, H. Kirk, M.D. (Indiv)
Compound
RT-100
Therapeutic Modality
Gene transfer
Target Gene
ADCY6
Vector Type
Adenovirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track
First Submitted: 2008-11-06
Last Modified Date: 1784610000000
Enrollment: 56
United States
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor
Beacon Therapeutics (Industry)
Compound
AGTC-0106
Therapeutic Modality
Gene transfer
Target Gene
SERPINA1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2010-01-21
Last Modified Date: 1784610000000
Enrollment: 9
Ireland, United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Jerry R. Mendell (Other)
Compound
RAAV1.CMV.huFollistatin344
Therapeutic Modality
Gene transfer
Target Gene
FST (FS344)
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2015-01-26
Last Modified Date: 1784610000000
Enrollment: 3
United States
Familial Lipoprotein Lipase Deficiency
Sponsor
Amsterdam Molecular Therapeutics (Industry)
Compound
GLYBERA
Therapeutic Modality
Gene transfer
Target Gene
LPL
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2009-04-30
Last Modified Date: 1784610000000
Enrollment: 5
Canada
Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor
Nationwide Children's Hospital (Other)
Compound
RAAV1.tMCK.human-alpha-sarcoglycan
Therapeutic Modality
Gene transfer
Target Gene
SGCA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2007-06-27
Last Modified Date: 1784610000000
Enrollment: 6
United States
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Lions Eye Institute, Perth, Western Australia (Other)
Compound
RAAV.sFlt-1
Therapeutic Modality
Gene transfer
Target Gene
Soluble VEGFR1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2011-12-14
Last Modified Date: 1784610000000
Enrollment: 40
Australia
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound
GALV MFGS-gc retrovirus
Therapeutic Modality
Gene transfer
Target Gene
IL2RG
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2001-12-17
Last Modified Date: 1784610000000
Enrollment: 3
United States
Hemophilia A
Sponsor
Spark Therapeutics, Inc. (Industry)
Compound
SPK-8016
Therapeutic Modality
Gene transfer
Target Gene
F8
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2018-11-06
Last Modified Date: 1784610000000
Enrollment: 4
United States
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor
Fondazione Telethon (Other)
Compound
STRIMVELIS
Therapeutic Modality
Gene transfer
Target Gene
ADA
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2008-01-10
Last Modified Date: 1784610000000
Enrollment: 12
Israel, Italy
Parotid Salivary Dysfunction
Sponsor
National Institute of Dental and Craniofacial Research (NIDCR) (NIH)
Compound
AdhAQP1
Therapeutic Modality
Gene transfer
Target Gene
AQP1
Vector Type
Adenovirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2006-09-02
Last Modified Date: 1784610000000
Enrollment: 17
United States
X-Linked Chronic Granulomatous Disease
Sponsor
University of Zurich (Other)
Compound
Retroviral SF71-gp91phox transduced CD34+ cells
Therapeutic Modality
Gene transfer
Target Gene
CYBB
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-06-22
Last Modified Date: 1784610000000
Enrollment: 2
Switzerland
Beta-Thalassemia Major
Sponsor
CorrectSequence Therapeutics Co., Ltd (Industry)
Compound
CS-101
Therapeutic Modality
Gene editing
Target Gene
BCL11A
Delivery System
Electroporation
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2024-02-23
Last Modified Date: 1781240400000
Enrollment: 9
China
Beta-Thalassemia Major
Sponsor
Sangamo Therapeutics (Industry)
Compound
ST-400
Therapeutic Modality
Gene editing
Target Gene
BCL11A
Delivery System
Electroporation
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Orphan Drug Designation
First Submitted: 2018-02-01
Last Modified Date: 1773723600000
Enrollment: 5
United States
Clinical Trials Daily Digest
Updates in the last 14 days
NCT05394064
1787720400000
With has results
Previous:
False
Current:
True
api
Last update post date
Previous:
2025-10-31
Current:
2026-08-26
api
Enrollment count
Previous:
8
Current:
9
api
Intervention description
Previous:
Sbt101 treatmentprocedure that mimics sbt101 infus...
Current:
Sbt101 treatment
api
Intervention name
Previous:
Sbt101, Imitation procedure
Current:
Sbt101 treatment
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. diagnosed with x-linked ad...
api
Adrenomyeloneuropathy (AMN)
SwanBio Therapeutics, Inc.
Terminated
Study was terminated for business/strategic reasons, not due to safety concerns
NCT07667387
1787720400000
Last update post date
Previous:
2026-08-11
Current:
2026-08-26
api
Estimated completion date
Previous:
2028-07
Current:
2028-09
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Brief title
Previous:
A phase i/ii open-label safety and efficacy study ...
Current:
Study of lnp.ucd.abe in patients with urea cycle d...
api
Eligibility criteria
Previous:
Inclusion criteria:
1. diagnosis of a severe urea...
Current:
Inclusion criteria:
1. diagnosis of a severe urea...
api
Record status
Previous:
Draft
Current:
Active
curator
Compound description
Previous:
Current:
personalized variant-specific LNP.UCD.ABE
curator
Indication doid
Previous:
Current:
DOID:9267
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
LNP.UCD.ABE
curator
Recent updates
Previous:
Current:
This clinical trial protocol is a master protocol ...
curator
Dose 1
Previous:
Current:
undisclosed dose based on body weight with potenti...
curator
Editor type
Previous:
Current:
ABE mRNA
curator
Delivery system
Previous:
Current:
Endocytosis
curator
Drug product type
Previous:
Current:
Encapsulated mRNA
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Mutation correction
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15
curator
Preclinical publications
Previous:
null
Current:
LinkName:Pre-IND Official Responses/Meeting Summar...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Pre-IND Briefing Book;LinkUrl:https://scg...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Patient-Specific In Vivo Gene Editing to ...
curator
Preclinical publications
Previous:
null
Current:
LinkName:How to create personalized gene editing p...
curator
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas
Recruiting
This clinical trial protocol is a master protocol for participants with a mutation in one of the 7 genes that cause a urea cycle disorder. Participant...
NCT03645460
1787720400000
Last update post date
Previous:
2026-06-23
Current:
2026-08-26
api
Estimated completion date
Previous:
2027-12-31
Current:
2028-12-31
api
Overall status
Previous:
Recruiting
Current:
Not yet recruiting
api
Vector type
Previous:
LV
Current:
Lentivirus
curator
Number of locations
Previous:
2
Current:
1
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* diagnosis of classical ada-...
api
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Shenzhen Geno-Immune Medical Institute
Not yet recruiting
NCT07287397
1787720400000
Last update post date
Previous:
2026-06-23
Current:
2026-08-26
api
Vector type
Previous:
AAV5.2
Current:
Adeno-associated virus
curator
Dose 2
Previous:
Undisclosed high dose
Current:
curator
Dose 1
Previous:
Undisclosed low dose
Current:
Undisclosed dose escalation, 2 levels
curator
Intervention description
Previous:
An investigational gene therapy targeting a specif...
Current:
An investigational gene therapy targeting a specif...
api
Intervention name
Previous:
Vtx-002, Optional rescue medication - corticostero...
Current:
Vtx-002, Preventative (prophylactic) medication - ...
api
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
1. capable of, And willin...
api
Record status
Previous:
Draft
Current:
Active
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) AAV-mediated intrabody therapy t...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) VecTabs® and advanced human in-v...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) AAV-mediated intrabody therapy t...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Non-clinical development and Pha...
curator
Indication doid
Previous:
null
Current:
DOID:332
curator
Development status
Previous:
null
Current:
Active
curator
Compound name
Previous:
null
Current:
VTx-002
curator
Recent updates
Previous:
null
Current:
FDA cleared IND 12/25
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Route of administration
Previous:
null
Current:
intracisternal
curator
Mechanism of action
Previous:
null
Current:
genetic delivery of therapeutic protein
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
antibody to TDP-43
curator
Fda designation
Previous:
Current:
Fast Track
curator
News and press releases
Previous:
null
Current:
LinkName:VectorY Therapeutics Receives FDA Clearan...
curator
News and press releases
Previous:
null
Current:
LinkName:VectorY Therapeutics Receives FDA Fast Tr...
curator
Amyotrophic Lateral Sclerosis
Vector Y Therapeutics
Recruiting
FDA cleared IND 12/25
NCT05230459
1787720400000
Last update post date
Previous:
2026-02-20
Current:
2026-08-26
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Enrollment type
Previous:
Estimated
Current:
Actual
api
Enrollment count
Previous:
10
Current:
9
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Dose 2
Previous:
Undisclosed dose 2
Current:
curator
Dose 1
Previous:
Undisclosed dose 1
Current:
Undisclosed dose escalation, 2 levels
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. male and female subjects a...
api
Limb-Girdle Muscular Dystrophy, Type 2I/R9
AskBio Inc
Active not recruiting
First patient dosed (8/3/23)
All Recent Updates (32)
NCT05394064
Show Changes
1787720400000
With has results
Previous:
False
Current:
True
api
Last update post date
Previous:
2025-10-31
Current:
2026-08-26
api
Enrollment count
Previous:
8
Current:
9
api
Intervention description
Previous:
Sbt101 treatmentprocedure that mimics sbt101 infus...
Current:
Sbt101 treatment
api
Intervention name
Previous:
Sbt101, Imitation procedure
Current:
Sbt101 treatment
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. diagnosed with x-linked ad...
api
Adrenomyeloneuropathy (AMN)
SwanBio Therapeutics, Inc.
Terminated
Study was terminated for business/strategic reasons, not due to safety concerns
NCT07667387
Show Changes
1787720400000
Last update post date
Previous:
2026-08-11
Current:
2026-08-26
api
Estimated completion date
Previous:
2028-07
Current:
2028-09
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Brief title
Previous:
A phase i/ii open-label safety and efficacy study ...
Current:
Study of lnp.ucd.abe in patients with urea cycle d...
api
Eligibility criteria
Previous:
Inclusion criteria:
1. diagnosis of a severe urea...
Current:
Inclusion criteria:
1. diagnosis of a severe urea...
api
Record status
Previous:
Draft
Current:
Active
curator
Compound description
Previous:
Current:
personalized variant-specific LNP.UCD.ABE
curator
Indication doid
Previous:
Current:
DOID:9267
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
LNP.UCD.ABE
curator
Recent updates
Previous:
Current:
This clinical trial protocol is a master protocol ...
curator
Dose 1
Previous:
Current:
undisclosed dose based on body weight with potenti...
curator
Editor type
Previous:
Current:
ABE mRNA
curator
Delivery system
Previous:
Current:
Endocytosis
curator
Drug product type
Previous:
Current:
Encapsulated mRNA
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Mutation correction
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15
curator
Preclinical publications
Previous:
null
Current:
LinkName:Pre-IND Official Responses/Meeting Summar...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Pre-IND Briefing Book;LinkUrl:https://scg...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Patient-Specific In Vivo Gene Editing to ...
curator
Preclinical publications
Previous:
null
Current:
LinkName:How to create personalized gene editing p...
curator
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas
Recruiting
This clinical trial protocol is a master protocol for participants with a mutation in one of the 7 genes that cause a urea cycle disorder. Participants with a variant that is amenable to correction by...
NCT03645460
Show Changes
1787720400000
Last update post date
Previous:
2026-06-23
Current:
2026-08-26
api
Estimated completion date
Previous:
2027-12-31
Current:
2028-12-31
api
Overall status
Previous:
Recruiting
Current:
Not yet recruiting
api
Vector type
Previous:
LV
Current:
Lentivirus
curator
Number of locations
Previous:
2
Current:
1
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* diagnosis of classical ada-...
api
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Shenzhen Geno-Immune Medical Institute
Not yet recruiting
NCT07287397
Show Changes
1787720400000
Last update post date
Previous:
2026-06-23
Current:
2026-08-26
api
Vector type
Previous:
AAV5.2
Current:
Adeno-associated virus
curator
Dose 2
Previous:
Undisclosed high dose
Current:
curator
Dose 1
Previous:
Undisclosed low dose
Current:
Undisclosed dose escalation, 2 levels
curator
Intervention description
Previous:
An investigational gene therapy targeting a specif...
Current:
An investigational gene therapy targeting a specif...
api
Intervention name
Previous:
Vtx-002, Optional rescue medication - corticostero...
Current:
Vtx-002, Preventative (prophylactic) medication - ...
api
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
1. capable of, And willin...
api
Record status
Previous:
Draft
Current:
Active
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) AAV-mediated intrabody therapy t...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) VecTabs® and advanced human in-v...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) AAV-mediated intrabody therapy t...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Non-clinical development and Pha...
curator
Indication doid
Previous:
null
Current:
DOID:332
curator
Development status
Previous:
null
Current:
Active
curator
Compound name
Previous:
null
Current:
VTx-002
curator
Recent updates
Previous:
null
Current:
FDA cleared IND 12/25
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Route of administration
Previous:
null
Current:
intracisternal
curator
Mechanism of action
Previous:
null
Current:
genetic delivery of therapeutic protein
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
antibody to TDP-43
curator
Fda designation
Previous:
Current:
Fast Track
curator
News and press releases
Previous:
null
Current:
LinkName:VectorY Therapeutics Receives FDA Clearan...
curator
News and press releases
Previous:
null
Current:
LinkName:VectorY Therapeutics Receives FDA Fast Tr...
curator
Amyotrophic Lateral Sclerosis
Vector Y Therapeutics
Recruiting
FDA cleared IND 12/25
NCT05230459
Show Changes
1787720400000
Last update post date
Previous:
2026-02-20
Current:
2026-08-26
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Enrollment type
Previous:
Estimated
Current:
Actual
api
Enrollment count
Previous:
10
Current:
9
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Dose 2
Previous:
Undisclosed dose 2
Current:
curator
Dose 1
Previous:
Undisclosed dose 1
Current:
Undisclosed dose escalation, 2 levels
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. male and female subjects a...
api
Limb-Girdle Muscular Dystrophy, Type 2I/R9
AskBio Inc
Active not recruiting
First patient dosed (8/3/23)
NCT05353647
Show Changes
1787720400000
Clinical publications
Previous:
null
Current:
LinkName:Long-term stability of posttranscriptiona...
curator
Last update post date
Previous:
2026-05-12
Current:
2026-08-25
api
Vector type
Previous:
VSV-G
Current:
Lentivirus
curator
Editor type
Previous:
none
Current:
curator
Estimated completion date
Previous:
2027-07
Current:
2027-10
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. a diagnosis of sickle cell...
api
Sickle Cell Disease
David Williams
Active not recruiting
NCT05139316
Show Changes
1787634000000
Compound description
Previous:
Current:
non-replicating, recombinant adeno-associated sero...
curator
Development status
Previous:
Active
Current:
Approved
curator
Compound name
Previous:
DTX401
Current:
GENGLYCOS
curator
Recent updates
Previous:
BLA Submission completed 12/30/25, PDUFA Action Da...
Current:
FDA approved on 8/20/26
curator
Dose 3
Previous:
Phase 3: 1.0E13 GC/kg
Current:
Approved dose: 1.0E13 GC/kg
curator
Dose 2
Previous:
Phase 1/2: 6E12 GC/kg
Current:
Phase 3: 1.0E13 GC/kg
curator
Dose 1
Previous:
Phase 1/2: 2E12 GC/kg
Current:
Phase 1/2 doses: 2E12 GC/kg, 6E12 GC/kg
curator
Alias type
Previous:
proper name
Current:
proper name, proprietary name
curator
Alias value
Previous:
Pariglasgene brecaparvovec
Current:
Pariglasgene brecaparvovec, DTX401
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Priority Revi...
Current:
Accelerated Approval, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Announces U.S. FDA Approval of...
curator
News and press releases
Previous:
null
Current:
LinkName:FDA Approval Documents;LinkUrl:https://ww...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) Top-Line Phase 3...
Current:
LinkName:(Corporate Presentation) August 2026;Link...
curator
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT03970278;LinkUrl:...
curator
Last update post date
Previous:
2026-03-11
Current:
2026-03-25
api
Estimated completion date
Previous:
2026-02-05
Current:
2026-02-20
api
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
* documented gsdia with c...
api
Clinical publications
Previous:
null
Current:
LinkName:Trial Interviews to Explore Glycogen Stor...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Reports Fourth Quarter and Ful...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Announces U.S. FDA Acceptance ...
curator
Overall status
Previous:
Active not recruiting
Current:
Completed
api
Glycogen Storage Disease Type Ia
Ultragenyx Pharmaceutical Inc
Completed
FDA approved on 8/20/26
NCT06884865
Show Changes
1787634000000
Last update post date
Previous:
2026-08-07
Current:
2026-08-25
api
Overall status
Previous:
Active not recruiting
Current:
Recruiting
api
Eligibility criteria
Previous:
50 years ii. morning stiffness \<30 minutes iii. crepitus on knee motion c. osteophytes
* subjects must have k-l grade 2, 3, Or 4 in the index knee based on x-rays performed during screening and confirmed by trained radiographers at a central facility before enrollment
* subjects need to show the presence of moderate or severe synovitis based on 11-point synovitis score using contrast-enhanced mri
exclusion criteria:
* subjects have any current or prior diagnosis of autoimmune connective tissue disorders, Secondary oa conditions, Benign synovial tumors, Gout/pseudogout, Reactive arthritis, Ra, Psoriatic arthritis, Ankylosing spondylitis, Or arthritis associated with inflammatory bowel disease.
* subjects have any active systemic or local infection, Including infection of the index knee
* subjects are unable to undergo mri with contrast mri
* subjects with x-ray or mri exclusionary events
* subjects have an unstable index knee joint (eg, Torn anterior cruciate ligament) within 12 months of screening
* subjects have used any approved or investigational ia drug/biologic in index knee within 6 months of screening (eg, Hyaluronic acid, Platelet rich plasma, Stem cells, Prolotherapy, And amniotic fluid injection)
* subjects are receiving or have received any gene therapy treatment (eg, Il-1ra) in the past 3 years
* subjects have used ia steroids ≤3 months before screening
other protocol-defined criteria apply">Inclusion criteria:
* subjects must be willing an...
Current:
50 years ii. morning stiffness \<30 minutes iii. crepitus on knee motion c. osteophytes
* subjects must have k-l grade 2, 3, Or 4 (part a) or k-l grade 2 or 3 (part b) in the index knee based on x-rays performed during screening and confirmed by trained radiographers at a central facility before enrollment
* subjects need to show the presence of moderate or severe synovitis based on 11-point synovitis score using contrast-enhanced mri (part a). for part b, Participants need to show the presence of synovitis ≥5 on the guermazi synovitis scoring scale using contrast-enhanced mri to be enrolled.
exclusion criteria:
* subjects have any current or prior diagnosis of autoimmune connective tissue disorders, Secondary oa conditions, Benign synovial tumors, Gout/pseudogout, Reactive arthritis, Ra, Psoriatic arthritis, Ankylosing spondylitis, Or arthritis associated with inflammatory bowel disease.
* subjects have any active systemic or local infection, Including infection of the index knee
* subjects are unable to undergo mri with contrast mri
* subjects with x-ray or mri exclusionary events
* subjects have an unstable index knee joint (eg, Torn anterior cruciate ligament) within 12 months of screening
* subjects have used any approved or investigational ia drug/biologic in index knee within 6 months of screening (eg, Hyaluronic acid, Platelet rich plasma, Stem cells, Prolotherapy, And amniotic fluid injection)
* subjects are receiving or have received any gene therapy treatment (eg, Il-1ra) in the past 3 years
* subjects have used ia steroids ≤3 months before screening
* for part b, Subjects with any surgery related tot he index knee.
other protocol-defined criteria apply">Inclusion criteria:
* subjects must be willing an...
api
Number of locations
Previous:
17
Current:
40
api
Vector type
Previous:
Ad5
Current:
Adenovirus
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intraarticular
Current:
Intra-articular
curator
Compound description
Previous:
null
Current:
AdV-NFKB-IL1RA
curator
Indication doid
Previous:
null
Current:
DOID:8398
curator
Development status
Previous:
null
Current:
Active
curator
Record status
Previous:
Provisional
Current:
Active
curator
Compound name
Previous:
null
Current:
PCRX-201
curator
Recent updates
Previous:
null
Current:
Pacira announced Phase 2 enrollment was complete i...
curator
Dose 3
Previous:
null
Current:
1.4E12 gc/knee
curator
Dose 2
Previous:
null
Current:
1.4E11 gc/knee
curator
Dose 1
Previous:
null
Current:
1.4E10 gc/knee
curator
Delivery system
Previous:
null
Current:
Viral transduction
curator
Drug product type
Previous:
null
Current:
Viral vector
curator
Mechanism of action
Previous:
null
Current:
Overexpression of protective allele/gene
curator
Therapy route
Previous:
null
Current:
In-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
IL1RA
curator
Alias type
Previous:
null
Current:
proper name, proprietary name
curator
Alias value
Previous:
null
Current:
Enekinragene inzadenovec, FX-201
curator
Fda designation
Previous:
Current:
Regenerative Medicine Advanced Therapy
curator
Preclinical publications
Previous:
null
Current:
LinkName:PCRX-201, a novel IL-1Ra gene therapy tre...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Efficacy and Safety of FX201, a Novel Int...
curator
News and press releases
Previous:
null
Current:
LinkName:Pacira BioSciences Announces PCRX-201 Gra...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) An Introduction ...
curator
News and press releases
Previous:
null
Current:
LinkName:Pacira BioSciences Concludes Patient Enro...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Abstract POS0492) A SINGLE INTRA-ARTICUL...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Abstract #594) Interim Data from the Fir...
curator
Estimated completion date
Previous:
2032-03
Current:
2032-07
api
Osteoarthritis (OA) of the Knee
Pacira Pharmaceuticals, Inc
Recruiting
Pacira announced Phase 2 enrollment was complete in November 2025
NCT03566043
Show Changes
1787634000000
Recent updates
Previous:
CRL issued 2/7/26 (issues with study eligibility c...
Current:
new spinal masses found in 5 patients dosed betwee...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) Rare Program Upd...
Current:
LinkName:(Corporate Presentation) Delivering the p...
curator
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Compound name
Previous:
RGX-121
Current:
NAVSUNLI
curator
Alias type
Previous:
proper name
Current:
proper name, proprietary name
curator
Alias value
Previous:
clemidosogene lanparvovec
Current:
clemidosogene lanparvovec, RGX-121
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Alignment with FDA on...
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracisterna magna
Current:
Intracisternal
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 3: NCT07236606;LinkUrl:https://clin...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
Eligibility criteria
Previous:
null
Current:
Part 1 inclusion criteria:
* the subject's legal ...
api
Mucopolysaccharidosis Type II (Hunter Syndrome)
REGENXBIO Inc.
Active not recruiting
New spinal masses found in 5 patients dosed between 3-6 years ago, clinical hold placed, REGENXBIO does not expect to resubmit the BLA in the near term
NCT07429240
Show Changes
1787634000000
Recent updates
Previous:
Initial data from multiple patients (target enroll...
Current:
First patient dosed in August 2026, Initial data f...
curator
Fda designation
Previous:
Fast Track
Current:
Fast Track, Orphan Drug Designation, Rare Pediatri...
curator
News and press releases
Previous:
null
Current:
LinkName:Precision BioSciences Commences Dosing in...
curator
Record status
Previous:
Draft
Current:
Active
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) ARCUS-Mediated Excision of Exons...
curator
Preclinical publications
Previous:
null
Current:
LinkName:High-efficiency homology-directed inserti...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Treatment with PBGENE-DMD result...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) PBGENE-DMD gene editing treatmen...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Presentation) PBGENE-DMD gene editing dr...
curator
Indication doid
Previous:
Current:
DOID:11723
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
PBGENE-DMD
curator
Dose 1
Previous:
Current:
1E14 vg/kg
curator
Editor type
Previous:
Current:
ARCUS endonuclease
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Drug product type
Previous:
Current:
Viral vector
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Exon skipping/splice editor
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
DMD del ex45-55
curator
News and press releases
Previous:
null
Current:
LinkName:Precision BioSciences Receives FDA Fast T...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) Precision BioSci...
curator
Last update post date
Previous:
2026-04-30
Current:
2026-06-23
api
Number of locations
Previous:
1
Current:
2
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Locations
Previous:
Current:
United states
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. males, 2 to 7 years of age...
api
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Precision BioSciences, Inc.
Recruiting
First patient dosed in August 2026, Initial data from multiple patients (target enrollment 3-5) anticipated by YE2026
NCT05248230
Show Changes
1787634000000
Compound description
Previous:
A101-CMV173-coCFTRΔR
Current:
A101-CMV173-coCFTRΔR(708-759)
curator
News and press releases
Previous:
LinkName:Corporate Presentation - July 2026;LinkUr...
Current:
LinkName:Corporate Presentation - August 2026;Link...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) Aerosolized 4D-7...
Current:
LinkName:(Corporate Presentation) 4D-710 for Cysti...
curator
Dose 4
Previous:
2E15 vg (discontinued due to high transduction to ...
Current:
curator
Dose 3
Previous:
1E15 vg (maximum tolerated dose)
Current:
curator
Dose 2
Previous:
5E14 vg
Current:
Phase 2 doses: 2.5E14 vg (anticipated pivotal/comm...
curator
Dose 1
Previous:
2.5E14 vg (anticipated pivotal/commercial dose)
Current:
Phase 1 doses: 1E15 vg (n=3), 2E15 vg (n=4, was di...
curator
Vector type
Previous:
AAV A101
Current:
Adeno-associated virus
curator
Last update post date
Previous:
2026-03-11
Current:
2026-07-06
api
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Inhalational
Current:
Respiratory (inhalation)
curator
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria (primary study):
1. 18 yea...
api
Estimated completion date
Previous:
2030-01
Current:
2031-06
api
Number of locations
Previous:
19
Current:
20
api
Enrollment count
Previous:
40
Current:
30
api
Cystic Fibrosis
4D Molecular Therapeutics
Recruiting
Interim data announced December 2025
NCT07064759
Show Changes
1787634000000
Clinical publications
Previous:
LinkName:(Corporate Presentation) July 2026;LinkUr...
Current:
LinkName:(Corporate Presentation) August 2026;Link...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) 4D-150 Business ...
Current:
LinkName:(Corporate Presentation) 4D-150 Business ...
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Recent updates
Previous:
4D-150 is also being evaluated for the treatment o...
Current:
Sponsor announced positive 2-year data from Phase ...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) 2-Year Follow-Up: PRISM P...
curator
News and press releases
Previous:
null
Current:
LinkName:Corporate Presentation July 2026;LinkUrl:...
curator
News and press releases
Previous:
null
Current:
LinkName:4DMT Announces Positive 2-Year Data from ...
curator
Editor type
Previous:
none
Current:
curator
Last update post date
Previous:
2026-02-12
Current:
2026-04-16
api
Number of locations
Previous:
43
Current:
94
api
Locations
Previous:
Argentina, Latvia, Singapore, Hungary, United stat...
Current:
Argentina, Singapore, Hungary, United states, Japa...
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* ≥50 years of age at time of...
api
Neovascular Age-Related Macular Degeneration (nAMD)
4D Molecular Therapeutics
Recruiting
Sponsor announced positive 2-year data from Phase 2b trial in July 2026; 4D-150 is also being evaluated for the treatment of Diabetic macular edema in a Phase 2 trial (NCT05930561)
NCT06680232
Show Changes
1787547600000
Last update post date
Previous:
2026-08-14
Current:
2026-08-24
api
Estimated completion date
Previous:
2026-12
Current:
2027-06
api
Number of locations
Previous:
4
Current:
7
api
Locations
Previous:
New zealand, Hong kong, United states, Moldova
Current:
New zealand, Romania, Hong kong, United states, Mo...
api
Vector type
Previous:
LNP
Current:
curator
Recent updates
Previous:
Cohort 5 dosing started at 0.65mg/kg, additional d...
Current:
New data readout in May 2026, Sponsor expected add...
curator
Dose 4
Previous:
0.65mg/kg
Current:
0.65mg/kg (n=4)
curator
Dose 3
Previous:
0.8 mg/kg
Current:
0.8 mg/kg (n=3)
curator
Dose 2
Previous:
0.4 mg/kg
Current:
0.4 mg/kg (n=6)
curator
Dose 1
Previous:
0.2mg/kg
Current:
0.2mg/kg (n=3)
curator
Target gene
Previous:
HBV DNA
Current:
HBV cccDNA
curator
News and press releases
Previous:
null
Current:
LinkName:Precision BioSciences Announces New and L...
curator
Preclinical publications
Previous:
null
Current:
LinkName:High-efficiency homology-directed inserti...
curator
Clinical publications
Previous:
null
Current:
LinkName:EASL 2026 Investor Update: PBGENE-HBV ELI...
curator
Delivery system
Previous:
Lipid encapsulation
Current:
Endocytosis
curator
Drug product type
Previous:
LNP
Current:
Encapsulated mRNA
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) First evidence of elimination an...
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT07254208;LinkUrl:...
curator
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
* male or women of non-ch...
api
News and press releases
Previous:
null
Current:
LinkName:Precision BioSciences Sets Strategic Prio...
curator
Chronic Hepatitis B
Precision BioSciences, Inc.
Recruiting
New data readout in May 2026, Sponsor expected additional updates by the end of 2026
NCT06063850
Show Changes
1787547600000
Last update post date
Previous:
2026-07-29
Current:
2026-08-24
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Recent updates
Previous:
First 3 patients in first cohort enrolled Septembe...
Current:
Preliminary data on first (low dose) cohort releas...
curator
Dose 2
Previous:
Current:
3E12 gc/ml
curator
Dose 1
Previous:
Undisclosed dose escalation, 2 levels
Current:
1E12 gc/ml
curator
News and press releases
Previous:
null
Current:
LinkName:uniQure Announces Preliminary Data on the...
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
Inclusion criteria:
* diagnosis of unilateral ref...
Current:
Inclusion criteria:
* diagnosis of unilateral ref...
api
Mesial Temporal Lobe Epilepsy
UniQure Biopharma B.V.
Recruiting
Preliminary data on first (low dose) cohort released June 2026, enrollment is ongoing in the second (high dose) cohort and is expected to be completed mid-2026
NCT01306019
Show Changes
1787547600000
Last update post date
Previous:
2026-08-18
Current:
2026-08-24
api
Description
Previous:
= 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, And who lack an hla-matched sibling donor. our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol.
prior successful retroviral gene transfer treatment instead of bone marrow transplant (bmt) in paris and london for 20 infants with xscid has provided proof of principle for efficacy. however, A major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of lmo2 and other dna regulatory genes by the strong enhancer present in the long-terminal repeat (ltr) of the moloney leukemia virus (mlv)- based vector.
furthermore, Previous studies of gene transfer treatment of older xscid patients with mlv- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected t- lymphocytes to the very high levels seen in infants. to reduce or eliminate this leukemia risk, And possibly enhance performance sufficiently to achieve benefit in older xscid patients, We have generated a lentivector with improved safety and performance features. we have generated a self-inactivating (sin) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (ef1a) internal promoter to expres...">This is a phase i/ii non-randomized clinical trial...
Current:
= 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, And who lack an hla-matched sibling donor. our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol.
prior successful retroviral gene transfer treatment instead of bone marrow transplant (bmt) in paris and london for 20 infants with xscid has provided proof of principle for efficacy. however, A major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of lmo2 and other dna regulatory genes by the strong enhancer present in the long-terminal repeat (ltr) of the moloney leukemia virus (mlv)- based vector.
furthermore, Previous studies of gene transfer treatment of older xscid patients with mlv- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected t- lymphocytes to the very high levels seen in infants. to reduce or eliminate this leukemia risk, And possibly enhance performance sufficiently to achieve benefit in older xscid patients, We have generated a lentivector with improved safety and performance features. we have generated a self-inactivating (sin) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (ef1a) internal promoter to expres......">This is a phase i/ii non-randomized clinical trial...
api
Vector type
Previous:
VSV-G
Current:
Lentivirus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
* inclusion criteria:
* a proven mutation in the c...
api
X-linked Severe Combined Immunodeficiency (XSCID)
National Institute of Allergy and Infectious Diseases (NIAID)
Recruiting
Developed in partnership with St. Jude, and licensed to Mustang Bio, Mustang Bio discontinued this program
NCT07113743
Show Changes
1787547600000
Last update post date
Previous:
2026-08-18
Current:
2026-08-24
api
Description
Previous:
Background:
x-linked chronic granulomatous diseas...
Current:
Background:
x-linked chronic granulomatous diseas...
api
Eligibility criteria
Previous:
2 x the upper limits of normal (uln) (patients with a correctable deficiency controlled on medication will not be excluded).
v. cytogenetic abnormalities known to be associated with hematopoietic defect on peripheral blood or bone marrow.
b. infectious
i. evidence of infection with hiv-1 and -2, Hepatitis b, Hepatitis c, Adenovirus, Parvovirus b 19 or toxoplasmosis within 8 weeks prior to mobilization/apheresis or bone marrow harvest. cytomegalovirus (cmv) infection is allowable as long as the infection is under control.
ii. history of infection with mycobacteria or bacille calmette-guerin (bcg) vaccination.
c. pulmonary
i. resting o2 saturation by pulse oximetry \< 90% on room air.
d. cardiac
i. abnormal electrocardiogram (ecg) indicating cardiac pathology.
ii. uncorrected congenital cardiac malformation with clinical symptomatology.
iii. active cardiac disease, Including clinical evidence of congestive heart failure, Cyanosis, Hypotension.
iv. poor cardiac function as evidenced by lv ejection fraction \<40% on echocardiogram.
e. neurological
i. significant neurologic abnormality by examination.
ii. uncontrolled seizure disorder.
f. renal
i. renal insufficiency: serum creatinine \>=2.5 mg/dl, Or \>=3+ proteinuria.
* chemistry lab abnormalities: serum sodium \>= 156 mmol/l or \<= 129 mmol/l, Potassium \>= 6.1 mmol/l or \<= 2.9 mmol/l, Calcium \>= 3.2 mmol/l or \< 1.74 mmol/l, Magnesium \>= 1.24 mmol/l or \< 0.39 mmol/l, Phosphate \>= 5.1 mmol/l or \< 1.9 mmol/l.
* serum transaminases \> 5x the upper limit of normal (uln).
serum bilirubin \> 2x the upper limit of normal (uln).
serum glucose \> 1.5x the upper limit of normal (uln).
* general
* expected survival \< 6 months.
* major congenital anomaly.
* known allergic reactions to components of busulfan or dimethyl sulfoxide (dmso) or contraindication for administration of conditioning medication.
* evidence of active malignant disease.
* treatment with another investigational drug or other intervention within 6 months.
* unable to undergo apheresis as per the nih cc department of transfusion medicine standard of care apheresis procedures.
1. patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm hg from the stable patient's baseline measurement) or requiring mechanical respiratory assistance are excluded.
history of vasculitis.
* administration of gamma-interferon within 30 days before the infusion of transduced, Autologous cd34+ cells.
* any other condition that, In the opinion of the investigator, May compromise the safety or compliance of the patient or would preclude the patient from successful study completion.">-inclusion criteria:
in order to be eligible to p...
Current:
2 x the upper limits of normal (uln) (patients with a correctable deficiency controlled on medication will not be excluded).
v. cytogenetic abnormalities known to be associated with hematopoietic defect on peripheral blood or bone marrow.
b. infectious
i. evidence of infection with hiv-1 and -2, Hepatitis b, Hepatitis c, Adenovirus, Parvovirus b 19 or toxoplasmosis within 8 weeks prior to mobilization/apheresis or bone marrow harvest. cytomegalovirus (cmv) infection is allowable as long as the infection is under control.
ii. history of infection with mycobacteria or bacille calmette-guerin (bcg) vaccination.
c. pulmonary
i. resting o2 saturation by pulse oximetry \< 90% on room air.
d. cardiac
i. abnormal electrocardiogram (ecg) indicating cardiac pathology.
ii. uncorrected congenital cardiac malformation with clinical symptomatology.
iii. active cardiac disease, Including clinical evidence of congestive heart failure, Cyanosis, Hypotension.
iv. poor cardiac function as evidenced by lv ejection fraction \<40% on echocardiogram.
e. neurological
i. significant neurologic abnormality by examination.
ii. uncontrolled seizure disorder.
f. renal
i. renal insufficiency: serum creatinine \>=2.5 mg/dl, Or \>=3+ proteinuria.
* chemistry lab abnormalities: serum sodium \>= 156 mmol/l or \<= 129 mmol/l, Potassium \>= 6.1 mmol/l or \<= 2.9 mmol/l, Calcium \>= 3.2 mmol/l or \< 1.74 mmol/l, Magnesium \>= 1.24 mmol/l or \< 0.39 mmol/l, Phosphate \>= 5.1 mmol/l or \< 1.9 mmol/l.
* serum transaminases \> 5x the upper limit of normal (uln).
serum bilirubin \> 2x the upper limit of normal (uln).
serum glucose \> 1.5x the upper limit of normal (uln).
* general
* expected survival \< 6 months.
* major congenital anomaly.
* known allergic reactions to components of busulfan or dimethyl sulfoxide (dmso) or contraindication for administration of conditioning medication.
* evidence of active malignant disease.
* treatment with another investigational drug or other intervention within 6 months.
* unable to undergo apheresis as per the nih cc department of transfusion medicine standard of care apheresis procedures.
1. patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm hg from the stable patient's baseline measurement) or requiring mechanical respiratory assistance are excluded.
history of vasculitis.
* administration of gamma-interferon within 21 days before the infusion of transduced, Autologous cd34+ cells.
* any other condition that, In the opinion of the investigator, May compromise the safety or compliance of the patient or would preclude the patient from successful study completion.">* inclusion criteria:
in order to be eligible to ...
api
Vector type
Previous:
Rhabdovirus
Current:
Lentivirus
curator
Editor type
Previous:
none
Current:
curator
Record status
Previous:
Draft
Current:
Active
curator
Preclinical publications
Previous:
null
Current:
LinkName:Non-Clinical Efficacy and Safety Studies ...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Lentiviral gene therapy for X-linked chro...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Biochemical correction of X-CGD by a nove...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Gene correction of induced pluripotent st...
curator
News and press releases
Previous:
null
Current:
LinkName:Orchard Therapeutics Announces FDA Grante...
curator
News and press releases
Previous:
null
Current:
LinkName:Orchard Therapeutics Extends Runway into ...
curator
Clinical publications
Previous:
null
Current:
LinkName:Lentiviral gene therapy for X-linked chro...
curator
Compound description
Previous:
Current:
pCCLChimGp91/VSVg lentiviral vector
curator
Indication doid
Previous:
Current:
DOID:0070195
curator
Development status
Previous:
Current:
Inactive
curator
Compound name
Previous:
Current:
VSVG-PCCLChimGp91
curator
Recent updates
Previous:
Current:
Commercial rights are owned by Orchard, which has ...
curator
Dose 1
Previous:
Current:
Transduced CD34+ cells (6.5-32.6E6 cells/kg)
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Target tissue
Previous:
Current:
CD34+ cells
curator
Drug product type
Previous:
Current:
Autologous cells
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Functional gene replacement
curator
Therapy route
Previous:
Current:
Ex-vivo
curator
Therapy type
Previous:
Current:
Gene transfer
curator
Target gene
Previous:
Current:
CYBB
curator
Alias value
Previous:
null
Current:
OTL-102
curator
Fda designation
Previous:
Current:
Orphan Drug Designation
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT02234934;LinkUrl:https://ww...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT02757911;LinkUrl:https://ww...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT01855685;LinkUrl:https://ww...
curator
Chronic Granulomatous Disease (CGD)
National Institute of Allergy and Infectious Diseases (NIAID)
Enrolling by invitation
Commercial rights are owned by Orchard, which has deprioritized the program
NCT02716246
Show Changes
1787288400000
Last update post date
Previous:
2026-07-17
Current:
2026-08-21
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Estimated completion date
Previous:
2027-07
Current:
2029-03
api
Recent updates
Previous:
BLA resubmitted 1/30/26, in February 2026 the Spon...
Current:
FDA has accepted the resubmitted BLA, and set a PD...
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Rare Pediatri...
Current:
Fast Track, Orphan Drug Designation, Priority Revi...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Announces U.S. FDA Acceptance ...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Resubmits Biologics License Ap...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Reports Fourth Quarter and Ful...
curator
Eligibility criteria
Previous:
Inclusion criteria:
* diagnosis of mps iiia confi...
Current:
Inclusion criteria:
* diagnosis of mps iiia confi...
api
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Ultragenyx Pharmaceutical Inc
Recruiting
FDA has accepted the resubmitted BLA, and set a PDUFA date of 9/19/26
NCT05930561
Show Changes
1787288400000
Last update post date
Previous:
2025-09-22
Current:
2026-08-21
api
Vector type
Previous:
AAV R100
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* ≥18 years of age
* type i o...
api
Diabetic Macular Edema, Diabetic Retinopathy
4D Molecular Therapeutics
Active not recruiting
52-week interim data update presented mid 2025
NCT07643844
Show Changes
1787288400000
Last update post date
Previous:
2026-06-23
Current:
2026-08-21
api
Vector type
Previous:
AAVrh10
Current:
Adeno-associated virus
curator
Record status
Previous:
Draft
Current:
Active
curator
Compound description
Previous:
Current:
AAVrh10-CAG-hPCCA
curator
Compound name
Previous:
Current:
AAVrh10-PCCA
curator
Dose 3
Previous:
Current:
3.2E13 vg/kg
curator
Dose 2
Previous:
Current:
8E12 vg/kg
curator
Dose 1
Previous:
Current:
2E12 vg/kg
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Drug product type
Previous:
Current:
Viral vector
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Functional gene replacement
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene transfer
curator
Target gene
Previous:
Current:
PCCA
curator
Preclinical publications
Previous:
null
Current:
LinkName:Long-term sex-biased correction of circul...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Effects of adeno-associated virus serotyp...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Review) Recent advances towards gene the...
curator
Indication doid
Previous:
Current:
DOID:14701
curator
Development status
Previous:
Current:
Active
curator
Fda designation
Previous:
Current:
Orphan Drug Designation, Rare Pediatric Disease De...
curator
News and press releases
Previous:
null
Current:
LinkName:Mayo researchers reveal gene therapy path...
curator
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Propionic Acidemia
Mayo Clinic
Recruiting
NCT05878860
Show Changes
1787288400000
Last update post date
Previous:
2026-08-10
Current:
2026-08-21
api
Number of locations
Previous:
18
Current:
19
api
Locations
Previous:
United states
Current:
United states, United kingdom
api
Vector type
Previous:
AAV.SPR
Current:
Adeno-associated virus
curator
News and press releases
Previous:
LinkName:
Atsena Therapeutics Doses First Patient...
Current:
LinkName:Atsena Therapeutics Doses First Patient a...
curator
Recent updates
Previous:
Enrollment in pivotal cohort is expected to comple...
Current:
First patient in pivotal cohort has been dosed. En...
curator
Editor type
Previous:
none
Current:
curator
Dose 5
Previous:
2.3E10 vg/eye (225ul)
Current:
curator
Dose 4
Previous:
1.5E10 vg/eye (150ul)
Current:
curator
Dose 3
Previous:
5.0E10 vg/eye
Current:
Phase 3: 1.1E10 vg/eye; n=39
curator
Dose 2
Previous:
3.0E10 vg/eye
Current:
Phase 1/2 Part B: 1.1E10 vg/eye, 1.7 vg/eye; n=3/c...
curator
Dose 1
Previous:
1.5E10 vg/eye
Current:
Phase 1/2 Part A: 1.1E10 vg/eye, 3.8E10 vg/eye, 2....
curator
Clinical publications
Previous:
LinkName:Safety and efficacy of ATSN-101 in patien...
Current:
null
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Safety and efficacy of ATS...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Safety and efficacy of ATS...
curator
News and press releases
Previous:
null
Current:
LinkName:Atsena Presents Positive Clinical Data fr...
curator
News and press releases
Previous:
LinkName:
Atsena Presents Positive Interim Six-Mo...
Current:
LinkName:Atsena Presents Positive Interim Six-Mont...
curator
News and press releases
Previous:
LinkName:
Atsena Therapeutics Receives Data Monit...
Current:
LinkName:Atsena Therapeutics Receives Data Monitor...
curator
Estimated completion date
Previous:
2029-10
Current:
2033-04
api
Official title
Previous:
A phase 1/2, Open-label, Dose escalation and dose ...
Current:
A phase 1/2/3, Open-label, Dose escalation, Dose e...
api
Eligibility criteria
Previous:
Inclusion criteria:
1. age ≥ 18 for cohorts 1 thr...
Current:
Part a and b:
inclusion criteria:
1. age ≥ 18 fo...
api
Eligibility sex
Previous:
Male
Current:
All
api
Enrollment count
Previous:
21
Current:
97
api
Phases
Previous:
Phase1, Phase2
Current:
Phase3
api
Description
Previous:
This study will evaluate the safety and tolerabili...
Current:
This study will evaluate the safety and efficacy o...
api
X-Linked Retinoschisis
Atsena Therapeutics Inc.
Recruiting
First patient in pivotal cohort has been dosed. Enrollment in pivotal cohort is expected to complete by end of Q1 2027, topline results expected 1H2028, BLA filing targeted for 2H2028
NCT05419492
Show Changes
1787288400000
Last update post date
Previous:
2026-08-19
Current:
2026-08-21
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Number of locations
Previous:
13
Current:
14
api
Dose 2
Previous:
Undisclosed high dose
Current:
curator
Dose 1
Previous:
Undisclosed low dose
Current:
Undisclosed dose escalation, 2 levels
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracerebroventricular
Current:
Intraventricular
curator
Fda designation
Previous:
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
Current:
Breakthrough Therapy, Chemistry, Manufacturing, an...
curator
Recent updates
Previous:
Received Breakthrough Designation from FDA in Janu...
Current:
Announced in May 2026 that the first patient in th...
curator
News and press releases
Previous:
null
Current:
LinkName:Encoded Therapeutics Doses First Patient ...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* participant must be aged be...
api
Dravet Syndrome
Encoded Therapeutics
Recruiting
Announced in May 2026 that the first patient in the pivotal trial has been dosed, initial data from Part 1B is expected in 4Q26
NCT05432310
Show Changes
1787202000000
Last update post date
Previous:
2026-04-07
Current:
2026-08-20
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Vector type
Previous:
LV
Current:
Lentivirus
curator
Editor type
Previous:
none
Current:
curator
Estimated completion date
Previous:
2026-12-31
Current:
2027-12-31
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
all subjects must fulfill the...
api
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
University of California, Los Angeles
Active not recruiting
Orchard terminated this program, returned the program to UCLA which administers the therapy under Compassionate Use
NCT07721025
Show Changes
1787115600000
News and press releases
Previous:
LinkName:(Corporate Presentation) Corporate Overvi...
Current:
LinkName:(Corporate Presentation) Corporate Overvi...
curator
Record status
Previous:
Draft
Current:
Active
curator
Compound description
Previous:
null
Current:
AAVrh.10-CAG-hFXN
curator
Indication doid
Previous:
null
Current:
DOID:0111218
curator
Development status
Previous:
null
Current:
Active
curator
Indication
Previous:
Friedreich Ataxia, Cardiomyopathy - Secondary
Current:
Friedreich Ataxia with Secondary Cardiomyopathy
curator
Compound name
Previous:
null
Current:
LX2006
curator
Recent updates
Previous:
null
Current:
Finalized pivotal trial protocol and statistical a...
curator
Dose 2
Previous:
null
Current:
Pivotal dose: 1.2E12 vg/kg
curator
Dose 1
Previous:
null
Current:
Dose escalation study: 1.8E11 vg/kg (n=6), 5.6E11 ...
curator
Vector type
Previous:
null
Current:
Adeno-associated virus
curator
Delivery system
Previous:
null
Current:
Viral transduction
curator
Drug product type
Previous:
null
Current:
Viral vector
curator
Route of administration
Previous:
null
Current:
Intravenous
curator
Mechanism of action
Previous:
null
Current:
Functional gene replacement
curator
Therapy route
Previous:
null
Current:
in vivo
curator
Therapy type
Previous:
null
Current:
Gene transfer
curator
Target gene
Previous:
null
Current:
FXN
curator
Fda designation
Previous:
Current:
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces Positive Int...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces Positive Int...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) LX2006 Regulator...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces Progress in ...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces FDA Breakthr...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Corporate Presentation) Lexeo Therapeuti...
curator
Clinical publications
Previous:
null
Current:
LinkName:AAVrh.10hFXN Gene Therapy for the Cardiom...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Expression and processing of mature human...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Stress-Induced Mouse Model of the Cardiac...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Identification of Safe and Effective Intr...
curator
Protocol
Previous:
null
Current:
LinkName:Clinical Trial Protocol;LinkUrl:https://c...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1: NCT05302271;LinkUrl:https://www....
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT05445323;LinkUrl:https://ww...
curator
Friedreich Ataxia with Secondary Cardiomyopathy
Lexeo Therapeutics
Recruiting
Finalized pivotal trial protocol and statistical analysis plan in June 2026, 1.2E12 will be pivotal dose, topline data readout expected 2H2027
NCT07218887
Show Changes
1787115600000
Last update post date
Previous:
2026-06-15
Current:
2026-08-19
api
Eligibility criteria
Previous:
Inclusion criteria:
* pathogenic or likely pathog...
Current:
Inclusion criteria:
* pathogenic or likely pathog...
api
Number of locations
Previous:
10
Current:
11
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Locations
Previous:
United states, Spain
Current:
United states, Italy, United kingdom, Spain
api
Dose 1
Previous:
Disclosed
Current:
undisclosed dose escalation, 3 levels
curator
Editor type
Previous:
none
Current:
curator
Indication doid
Previous:
null
Current:
DOID:0110448
curator
Development status
Previous:
null
Current:
Active
curator
Record status
Previous:
Provisional
Current:
Active
curator
Compound name
Previous:
null
Current:
ALXN2350
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Route of administration
Previous:
null
Current:
intravenous
curator
Mechanism of action
Previous:
null
Current:
functional gene replacement
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
BAG3
curator
Related nctid
Previous:
null
Current:
LinkName:Natural History Study: NCT05981092;LinkUr...
curator
BAG3 Mutation Associated Dilated Cardiomyopathy
Alexion Pharmaceuticals, Inc.
Recruiting
NCT04370054
Show Changes
1787115600000
Last update post date
Previous:
2026-05-18
Current:
2026-08-19
api
Dose 5
Previous:
Phase 3: 3E13 vg/kg (n=75)
Current:
curator
Dose 4
Previous:
Phase 1/2: 3E13 vg/kg (n=5)
Current:
curator
Dose 3
Previous:
Phase 1/2: 1E13 vg/kg (n=2)
Current:
curator
Dose 2
Previous:
Phase 1/2: 2E12 vg/kg (n=2)
Current:
Phase 3: 3E13 vg/kg (n=75)
curator
Dose 1
Previous:
Phase 1/2: 9E11 vg/kg (n=2)
Current:
Phase 1/2: 9E11 vg/kg (n=2); 2E12 vg/kg (n=2); 1E1...
curator
Vector type
Previous:
AAV2/6
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Estimated completion date
Previous:
2028-10-25
Current:
2026-10-25
api
Number of locations
Previous:
36
Current:
45
api
Eligibility criteria
Previous:
null
Current:
Main inclusion criteria
* males who have been fol...
api
Hemophilia A
Pfizer
Active not recruiting
Pfizer terminated this program due to marketing considerations, Development rights are being returned to Sangamo
NCT04671433
Show Changes
1787115600000
Recent updates
Previous:
Phase 3 dosing complete, phase 3 study did not mee...
Current:
Phase 3 dosing complete, phase 3 study did not mee...
curator
News and press releases
Previous:
LinkName:Corporate Presentation - June 2026;LinkUr...
Current:
LinkName:Corporate Presentation - August 2026;Link...
curator
Vector type
Previous:
AAV2/5
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT04671433;LinkUrl:...
curator
Last update post date
Previous:
2025-09-29
Current:
2026-03-16
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* male or female
* 3 years of...
api
Number of locations
Previous:
27
Current:
28
api
Enrollment count
Previous:
97
Current:
105
api
X-Linked Retinitis Pigmentosa (XLRP)
Janssen Research & Development, LLC
Completed
Phase 3 dosing complete, phase 3 study did not meet its primary efficacy endpoint but showed clinical meaningful benefits in multiple secondary endpoints
NCT06109181
Show Changes
1787115600000
News and press releases
Previous:
LinkName:(Corporate Presentation) Corporate Overvi...
Current:
LinkName:(Corporate Presentation) Corporate Overvi...
curator
Vector type
Previous:
AAVrh10
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Selected inclusion criteria:
* adults with a clin...
api
Arrhythmogenic Right Ventricular Cardiomyopathy
Lexeo Therapeutics
Active not recruiting
Completed enrollment of LX2020 Phase 1/2 trial, additional clinical data expected January 2026
NCT04411654
Show Changes
1787115600000
Last update post date
Previous:
2026-04-01
Current:
2026-08-19
api
Estimated completion date
Previous:
2028-05
Current:
2027-02
api
Number of locations
Previous:
5
Current:
6
api
Recent updates
Previous:
Current:
Lilly acquired this company in 2020, announced the...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) Q2 2026 Earnings...
curator
Preclinical publications
Previous:
LinkName:(Abstract #623) Design of a Phase 1/2 Stu...
Current:
LinkName:(Abstract #623) Design of a Phase 1/2 Stu...
curator
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Dose 2
Previous:
Undisclosed high dose
Current:
curator
Dose 1
Previous:
Undisclosed low dose
Current:
Undisclosed dose escalation, 2 levels
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracisterna magna
Current:
Intracisternal
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* bi-allelic gba1 mutations c...
api
Gaucher Disease Type 2
Prevail Therapeutics
Active not recruiting
Lilly acquired this company in 2020, announced they were dropping this asset in August of 2026
NCT06332807
Show Changes
1787115600000
Last update post date
Previous:
2025-12-02
Current:
2026-08-18
api
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. is willing and able to pro...
api
Phenylketonuria (PKU)
NGGT INC.
Recruiting
Recieved Orphan Drug Designation from the FDA in Jan 2023
NCT05606614
Show Changes
1787115600000
News and press releases
Previous:
LinkType:Clinical Publications;LinkName:(Corporate...
Current:
LinkType:News and Press Releases;LinkName:Taysha G...
curator
Compound description
Previous:
Current:
scAAV-MeP426-miniMECP2-miRARE
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) rAAV9 Vector Biodistribution in ...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Every Gain, Expanding Possibilit...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) REVEAL Adolescent/Adult and Pedi...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Achieving Developmental Mileston...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster #54) Superior expression of self-...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster #7) Establishing the Rett Syndrom...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster #39) The Developmental Plateau in...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster #59) Safety and Efficacy Results ...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) TSHA-102 in clin...
Current:
LinkName:(Corporate Presentation) TSHA-102 Rett Sy...
curator
Vector type
Previous:
scAAV9
Current:
Adeno-associated virus
curator
Last update post date
Previous:
2025-12-30
Current:
2026-07-07
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Number of locations
Previous:
6
Current:
9
api
Enrollment type
Previous:
Estimated
Current:
Actual
api
Enrollment count
Previous:
15
Current:
17
api
Recent updates
Previous:
First patient dosed in REVEAL pivotal trial (adole...
Current:
Dosing completed in pivotal trial, topline data an...
curator
News and press releases
Previous:
null
Current:
LinkName:Taysha Gene Therapies Announces Completio...
curator
Editor type
Previous:
none
Current:
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 3: NCT07480564 (Pediatric cohort);L...
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Rare Pediatri...
Current:
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:Taysha Gene Therapies Announces Progress ...
curator
News and press releases
Previous:
null
Current:
LinkName:SEC Form 10-K: Taysha Gene Therapies, Inc...
curator
News and press releases
Previous:
null
Current:
LinkName:Taysha Gene Therapies Regains Full Rights...
curator
Record status
Previous:
Inactive
Current:
Active
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* females between the ages of...
api
Rett Syndrome
Taysha Gene Therapies, Inc.
Active not recruiting
Dosing completed in pivotal trial, topline data and FDA feedback on BLA submission pathway expected 1H 2027, BLA submission expected 1H 2027
NCT06831825
Show Changes
1786942800000
Sponsor
Previous:
Yap therapeutics, Inc.
Current:
Medley therapeutics
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Target tissue
Previous:
Cardiomyocyte
Current:
Myocardium
curator
Route of administration
Previous:
Transendocardial
Current:
Intracardiac
curator
Preclinical publications
Previous:
null
Current:
LinkName:Gene therapy against Hippo triggers cardi...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Hippo pathway deficiency reverses systoli...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
to participate, A subject mus...
api
Heart Failure With Reduced Ejection Fraction
Medley Therapeutics
Recruiting
First cohort (n=3) dosing is completed
NCT07180355
Show Changes
1786942800000
Last update post date
Previous:
2026-07-30
Current:
2026-08-17
api
Vector type
Previous:
AAVhu68
Current:
Adeno-associated virus
curator
Target tissue
Previous:
Current:
Dentate nucleus
curator
Route of administration
Previous:
Intradentate nucleus, intravenous
Current:
Intraparenchymal, Intravenous
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* has history of fa symptom o...
api
Record status
Previous:
Draft
Current:
Active
curator
Indication doid
Previous:
null
Current:
DOID:0111218
curator
Development status
Previous:
null
Current:
Active
curator
Indication
Previous:
Friedreich's Ataxia (FA)
Current:
Friedreich's Ataxia
curator
Compound name
Previous:
null
Current:
SGT-212
curator
Recent updates
Previous:
null
Current:
first patient dosed January 2026, initial data exp...
curator
Dose 1
Previous:
null
Current:
undisclosed
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Mechanism of action
Previous:
null
Current:
functional gene replacement
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
FXN
curator
Fda designation
Previous:
Current:
Fast Track, Orphan Drug Designation, Rare Pediatri...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) January 2026;Lin...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Efficacy and Safety of a Novel A...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Receives FDA Orphan Dru...
curator
Friedreich's Ataxia
Solid Biosciences Inc.
Recruiting
First patient dosed January 2026, initial data expected H2 2026
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.
Disclaimer
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.