SCGE Platform - Gene Therapy Clinical Trials

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, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

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.

Updates in the last 14 days

NCT07282847

2026-05-28

Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD

Recruiting

FDA accepted IND in January 2026

NCT06461702

2026-05-28

Familial Hypercholesterolemia

Unknown

FDA cleared IND application in June 2025

NCT06856759

2026-05-28

Rett Syndrome

Active not recruiting

NCT07052929

2026-05-28

X-Linked Myotubular Myopathy

Recruiting

Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver,...

NCT07592273

2026-05-28

Diabetic Retinopathy

Recruiting

This product is also under evaluation for wet AMD

Showing 1-57 of 57 results in ClinicalTrials

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NCT07592273 Recruiting Phase3, Phase2

View Report

Diabetic Retinopathy

Sponsor AbbVie (Industry)

Compound Surabgene lomparvovec

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05968118 Unknown Phase3

View Report

Peripheral Artery Disease

Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)

Compound NL003

Therapy Type Gene transfer

Target Gene HGF

Mechanism of Action Overexpression of protective allele/gene

NCT04275323 Completed Phase3

View Report

Critical Limb Ischemia

Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)

Compound NL003

Therapy Type Gene transfer

Target Gene HGF

Mechanism of Action Overexpression of protective allele/gene

NCT02563522 Terminated Phase3

View Report

Diabetic Foot Ulcer

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapy Type Gene transfer

Target Gene HGF

Mechanism of Action Overexpression of protective allele/gene

NCT07007065 Recruiting Phase3

View Report

Neovascular Age-related Macular Degeneration

Sponsor AbbVie (Industry)

Compound ABBV-RGX-314

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT02144610 Terminated Phase3

View Report

Critical Limb Ischemia

Sponsor AnGes USA, Inc. (Industry)

Compound COLLATEGENE

Therapy Type Gene transfer

Target Gene HGF

Mechanism of Action Overexpression of protective allele/gene

NCT06128629 Recruiting Phase3

View Report

Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

Sponsor Intellia Therapeutics (Industry)

Compound NTLA-2001

Therapy Type Gene editing

Target Gene TTR

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT04469270 Completed Phase3

View Report

Diabetic Neuropathy

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapy Type Gene transfer

Target Gene HGF

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT06634420 Active not recruiting Phase3

View Report

Hereditary Angioedema

Sponsor Intellia Therapeutics (Industry)

Compound NTLA-2002

Therapy Type Gene editing

Target Gene KLKB1

Vector Type LDLR

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT05881408 Active not recruiting Phase3

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Sarepta Therapeutics, Inc. (Industry)

Compound ELEVIDYS

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAVrh74

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06856577 Active not recruiting Phase3

View Report

Neovascular (Wet) Age-Related Macular Degeneration (nAMD)

Sponsor Adverum Biotechnologies, Inc. (Industry)

Compound ADVM-022

Therapy Type Gene transfer

Target Gene Codon optimized aflibercept

Vector Type AAV.7m8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track

NCT04370054 Active not recruiting Phase3

View Report

Hemophilia A

Sponsor Pfizer (Industry)

Compound Giroctogogene fitelparvovec

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV2/6

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation

NCT03861273 Active not recruiting Phase3

View Report

Hemophilia B

Sponsor Pfizer (Industry)

Compound BEQVEZ

Therapy Type Gene transfer

Target Gene F9-R338L

Vector Type AAVrh74

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT07160634 Recruiting Phase3

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-003

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV-SLB101

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT05878860 Recruiting Phase3

View Report

X-Linked Retinoschisis

Sponsor Atsena Therapeutics Inc. (Industry)

Compound ATSN-201

Therapy Type Gene transfer

Target Gene RS1

Vector Type AAV.SPR

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02716246 Recruiting Phase3, Phase2

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX111

Therapy Type Gene transfer

Target Gene SGSH

Vector Type AAV9

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

NCT06199531 Active not recruiting Phase3

View Report

NGLY1 Deficiency

Sponsor Grace Science, LLC (Industry)

Compound GS-100

Therapy Type Gene transfer

Target Gene NGLY1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04704921 Active not recruiting Phase3, Phase2

View Report

Neovascular (Wet) Age-Related Macular Degeneration (nAMD)

Sponsor AbbVie (Industry)

Compound ABBV-RGX-314

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06692712 Recruiting Phase3

View Report

Hereditary Spastic Paraplegia Type 50

Sponsor Elpida Therapeutics SPC (Industry)

Compound MELPIDA

Therapy Type Gene transfer

Target Gene AP4M1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07223944 Recruiting Phase3

View Report

Gaucher Disease Type 1

Sponsor Spur Therapeutics (Industry)

Compound FLT201

Therapy Type Gene transfer

Target Gene GBA1

Vector Type AAVS3

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT06432140 Active not recruiting Phase3, Phase2

View Report

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)

Compound VGN-R09b

Therapy Type Gene transfer

Target Gene DDC

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07064759 Recruiting Phase3

View Report

Neovascular Age-Related Macular Degeneration (nAMD)

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-150

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT03199469 Active not recruiting Phase3, Phase2

View Report

X-Linked Myotubular Myopathy

Sponsor Astellas Gene Therapies (Industry)

Compound AT132

Therapy Type Gene transfer

Target Gene MTM1

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05386680 Completed Phase3

View Report

Spinal Muscular Atrophy

Sponsor Novartis Pharmaceuticals (Industry)

Compound ITVISMA

Therapy Type Gene transfer

Target Gene SMN1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Orphan Drug Designation

NCT04850118 Active not recruiting Phase3, Phase2

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-501

Therapy Type Gene transfer

Target Gene RPGR

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation

NCT03566043 Active not recruiting Phase3, Phase2

View Report

Mucopolysaccharidosis Type II (Hunter Syndrome)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-121

Therapy Type Gene transfer

Target Gene IDS

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05606614 Recruiting Phase3

View Report

Rett Syndrome

Sponsor Taysha Gene Therapies, Inc. (Industry)

Compound TSHA-102

Therapy Type Gene transfer

Target Gene MiniMECP2

Vector Type scAAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05345171 Active not recruiting Phase3

View Report

Ornithine Transcarbamylase (OTC) Deficiency

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound DTX301

Therapy Type Gene transfer

Target Gene OTC

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT04293185 Active not recruiting Phase3

View Report

Sickle Cell Disease

Sponsor Genetix Biotherapeutics Inc. (Industry)

Compound LYFGENIA

Therapy Type Gene transfer

Target Gene HBB

Vector Type BB305 LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04671433 Completed Phase3

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Janssen Research & Development, LLC (Industry)

Compound Botaretigene sparoparvovec

Therapy Type Gene transfer

Target Gene RPGR

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05956626 Active not recruiting Phase3, Phase2

View Report

Stargardt Disease

Sponsor Ocugen (Industry)

Compound OCU410ST

Therapy Type Gene transfer

Target Gene RORA

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT05139316 Completed Phase3

View Report

Glycogen Storage Disease Type Ia

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound DTX401

Therapy Type Gene transfer

Target Gene G6PC

Vector Type AAV8

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

NCT06388200 Active not recruiting Phase3

View Report

Retinitis Pigmentosa

Sponsor Ocugen (Industry)

Compound OCU400

Therapy Type Gene transfer

Target Gene NR2E3

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT05725018 Active not recruiting Phase3

View Report

Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Sponsor Abeona Therapeutics, Inc (Industry)

Compound ZEVASKYN

Therapy Type Gene transfer

Target Gene COL7A1

Vector Type LZRSE

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT00999609 Active not recruiting Phase3

View Report

Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis

Sponsor Spark Therapeutics, Inc. (Industry)

Compound LUXTURNA

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02652767 Completed Phase3

View Report

Leber Hereditary Optic Neuropathy (LHON)

Sponsor GenSight Biologics (Industry)

Compound LUMEVOQ

Therapy Type Gene transfer

Target Gene ND4G11778A

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03496012 Completed Phase3

View Report

Choroideremia

Sponsor Biogen (Industry)

Compound BIIB111

Therapy Type Gene transfer

Target Gene CHM

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT05203679 Active not recruiting Phase3, Phase2

View Report

Hemophilia B

Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)

Compound BBM-H901

Therapy Type Gene transfer

Target Gene F9

Vector Type AAV843

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03569891 Completed Phase3

View Report

Hemophilia B

Sponsor CSL Behring (Industry)

Compound HEMGENIX

Therapy Type Gene transfer

Target Gene F9-R338L

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation

NCT05477563 Recruiting Phase3

View Report

Beta-Thalassemia, Sickle Cell Disease

Sponsor Vertex Pharmaceuticals Incorporated (Industry)

Compound CASGEVY

Therapy Type Gene editing

Target Gene BCL11A

Vector Type RNP

Delivery System Electroporation

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05898620 Recruiting Phase3

View Report

Rett Syndrome

Sponsor Neurogene Inc. (Industry)

Compound NGN-401

Therapy Type Gene transfer

Target Gene MECP2

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03837483 Active not recruiting Phase3

View Report

Wiskott-Aldrich Syndrome

Sponsor Fondazione Telethon (Other)

Compound WASKYRA

Therapy Type Gene transfer

Target Gene WAS

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06149403 Active not recruiting Phase3

View Report

Mucopolysaccharidosis Type I (Hurler Syndrome)

Sponsor Orchard Therapeutics (Industry)

Compound OTL-203

Therapy Type Gene transfer

Target Gene IDUA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04917874 Completed Phase3

View Report

Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Sponsor Krystal Biotech, Inc. (Industry)

Compound VYJUVEK

Therapy Type Gene transfer

Target Gene COL7A1

Vector Type HSV1

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

NCT04281485 Active not recruiting Phase3

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Pfizer (Industry)

Compound PF-06939926

Therapy Type Gene transfer

Target Gene Mini-dystrophin

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06111638 Recruiting Phase3, Phase2

View Report

Hemophilia A

Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)

Compound BBM-H803

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT04323098 Completed Phase3

View Report

Hemophilia A

Sponsor BioMarin Pharmaceutical (Industry)

Compound ROCTAVIAN

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation

NCT04283227 Active not recruiting Phase3

View Report

Metachromatic Leukodystrophy

Sponsor Orchard Therapeutics (Industry)

Compound LENMELDY

Therapy Type Gene transfer

Target Gene ARSA

Vector Type VSV-G

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06246513 Active not recruiting Phase3

View Report

Limb-Girdle Muscular Dystrophy, Type 2E/R4

Sponsor Sarepta Therapeutics, Inc. (Industry)

Compound SRP-9003

Therapy Type Gene transfer

Target Gene SGCB

Vector Type AAVrh74

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03612869 Unknown Phase3, Phase2

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor LYSOGENE (Industry)

Compound LYS-SAF301

Therapy Type Gene transfer

Target Gene SGSH

Vector Type AAV2rh.10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06833983 Recruiting Phase3

View Report

Hemophilia A

Sponsor Gritgen Therapeutics Co., Ltd. (Industry)

Compound GS1191-0445

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03207009 Completed Phase3

View Report

Beta-Thalassemia Major

Sponsor Genetix Biotherapeutics Inc. (Industry)

Compound ZYNTEGLO

Therapy Type Gene transfer

Target Gene HBB

Vector Type BB305 LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06699108 Active not recruiting Phase3

View Report

Bietti Crystalline Dystrophy

Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)

Compound VGR-R01

Therapy Type Gene transfer

Target Gene CYP4V2

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT00891306 Completed Phase3, Phase2

View Report

Familial Lipoprotein Lipase Deficiency

Sponsor Amsterdam Molecular Therapeutics (Industry)

Compound GLYBERA

Therapy Type Gene transfer

Target Gene LPL

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05693142 Recruiting Phase3, Phase2

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-202

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06743646 Recruiting Phase3

View Report

Bietti Crystalline Dystrophy

Sponsor Chigenovo Co., Ltd (Network)

Compound ZVS101e

Therapy Type Gene transfer

Target Gene CYP4V2

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03852498 Completed Phase3

View Report

Cerebral Adrenoleukodystrophy (CALD)

Sponsor Genetix Biotherapeutics Inc. (Industry)

Compound SKYSONA

Therapy Type Gene transfer

Target Gene ABCD1

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation

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

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.

Industry	All for-profit entities
NIH	U.S. National Institutes of Health
Other Non-Profit	Includes individuals, universities, community-based organizations

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

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

Clinical Trials - Gene Therapy Trial Browser

Clinical Trials Daily Digest

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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.

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

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)

Ex-vivo	When the cells are modified outside the body
In-vivo	When the cells are modified inside the body

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

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