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.
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Showing all 51 results in ClinicalTrials
SCGE Platform Gene Therapy Clinical Trials downloaded on: 2025/12/16 10:05:36; 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
Product was acquired by uniQure, uniQure did not renew their EMA marketing application when it expired 10/25/17, and Glybera is no longer commercially available
Dose 1: Total dose: 4mg or 8mg or 16mg (injections divided on day 0 and day 14) | Dose 2: Total dose: 16mg/32 x 0.5ml injections/gastrocnemius | Dose 3: Total dose: 32mg/32 x 0.5ml injections/gastrocnemius | Dose 4: Total dose: 32mg, concentration: 0.25mg/0.5ml, 16injections/day, 4 days: 0, 14 ,90, 104, bilateral gastroc.
Support for Clinical Trials Advancing Rare Disease Therapeutics, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
NGN-401
Neurogene Inc.
Industry
MECP2
Gene transfer
In-vivo
Functional gene replacement
Intracerebroventricular
Viral vector
Viral transduction
AAV9
Dose 1: 1E15 vg | Dose 2: 3E15 vg (discontinued after patient death due to HLH)
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), United Kingdom, United States
WO2017074526
Pfizer terminated this program due to marketing considerations, Development rights are being returned to Sangamo
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), United Kingdom, United States
FDA approved 4/25/24, Price/treatment $3.5M; Pfizer will no longer market Beqvez
Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom, United States
US20230173101A1
First approval was June 2023, expanded indication to all DMD patients regardless of ambulatory status or age on 6/20/24; new black box warning on risk of liver injury and liver failure added November 2025
Belgium, Germany, Italy, Spain, United Kingdom, United States
US20230241252A1; WO2021257595A1; EP4219726A1
FDA placed clinical hold in July 2025 due to safety concerns with the AAV after the death of a patient who had received SRP-9004, which uses the same AAV vector, the Company plans to meet with FDA regarding BLA submission in 2025
BLA filed under Accelerated Approval, CRL issued 7/11/25 requesting additional information and improvements related to CMC and manufacturing inspections