^
    17h
    GALILEO-3: A Gaucher Disease Gene Therapy Trial With FLT201 (clinicaltrials.gov)
    P3, N=45, Recruiting, Spur Therapeutics | Not yet recruiting --> Recruiting
    Enrollment open
    20h
    A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency (clinicaltrials.gov)
    P2/3, N=13, Active, not recruiting, Shanghai Vitalgen BioPharma Co., Ltd. | Phase classification: P3 --> P2/3
    Phase classification
    5d
    A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) (clinicaltrials.gov)
    P1/2, N=26, Recruiting, Aspa Therapeutics | Trial completion date: Oct 2030 --> Oct 2032
    Trial completion date
    6d
    A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) (clinicaltrials.gov)
    P1, N=9, Recruiting, Rocket Pharmaceuticals Inc. | Trial completion date: Sep 2026 --> Sep 2029 | Trial primary completion date: Sep 2025 --> Sep 2028
    Trial completion date • Trial primary completion date
    7d
    OTC-HOPE: An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (clinicaltrials.gov)
    P1/2, N=20, Recruiting, iECURE, Inc. | Trial completion date: Sep 2026 --> Dec 2027 | Trial primary completion date: Sep 2026 --> Dec 2027 | Active, not recruiting --> Recruiting
    Enrollment open • Trial completion date • Trial primary completion date • First-in-human
    7d
    BEYOND-9: A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B (clinicaltrials.gov)
    P1/2, N=130, Recruiting, Regeneron Pharmaceuticals | Trial completion date: Dec 2032 --> Aug 2047 | Trial primary completion date: Dec 2032 --> Aug 2034
    Trial completion date • Trial primary completion date
    8d
    Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 (clinicaltrials.gov)
    P=N/A, N=14, Enrolling by invitation, Rocket Pharmaceuticals Inc.
    New trial
    |
    FANCA (FA Complementation Group A)
    9d
    PRISM: Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection) (clinicaltrials.gov)
    P1/2, N=21, Not yet recruiting, Zhongmou Therapeutics | Initiation date: Feb 2026 --> Jun 2026
    Trial initiation date
    9d
    A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (clinicaltrials.gov)
    P3, N=4, Active, not recruiting, Janssen Pharmaceutical K.K. | Recruiting --> Active, not recruiting
    Enrollment closed
    10d
    OTC-HOPE: An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency (clinicaltrials.gov)
    P1/2, N=8, Active, not recruiting, iECURE, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed • First-in-human
    15d
    A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP (clinicaltrials.gov)
    P2/3, N=85, Active, not recruiting, Beacon Therapeutics | Trial completion date: Oct 2029 --> Oct 2031 | Trial primary completion date: Aug 2025 --> Jun 2026
    Trial completion date • Trial primary completion date
    16d
    Guardian: A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease (clinicaltrials.gov)
    P2/3, N=51, Active, not recruiting, Ocugen | Recruiting --> Active, not recruiting
    Enrollment closed