^
    2d
    ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) (clinicaltrials.gov)
    P4, N=36, Not yet recruiting, Novartis Pharmaceuticals
    New P4 trial • Real-world evidence
    4d
    REVISE: Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients (clinicaltrials.gov)
    P2, N=14, Recruiting, GenSight Biologics | Not yet recruiting --> Recruiting
    Enrollment open
    7d
    AAVIATE: RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD) (clinicaltrials.gov)
    P2, N=146, Active, not recruiting, AbbVie | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    Lucentis (ranibizumab)
    7d
    RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) (clinicaltrials.gov)
    P3, N=2, Suspended, REGENXBIO Inc. | Recruiting --> Suspended
    Trial suspension
    10d
    Long Term Follow-up for RGX-202 (clinicaltrials.gov)
    P=N/A, N=66, Enrolling by invitation, REGENXBIO Inc. | N=19 --> 66
    Enrollment change
    12d
    J3Z-MC-OJAE: A dose finding study of LY3884961 in patients with Gaucher disease (2022-500281-10-02)
    P1/2, N=6, Active, not recruiting, Prevail Therapeutics Inc. | Suspended --> Active, not recruiting
    Enrollment closed
    |
    prednisone • sirolimus • methylprednisolone sodium succinate
    14d
    Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION) (clinicaltrials.gov)
    P1, N=10, Active, not recruiting, Ray Therapeutics, Inc. | N=18 --> 10 | Recruiting --> Active, not recruiting
    Enrollment closed • Enrollment change
    15d
    A Biomarker Study in Men With Localized Prostate Cancer Treated With Aglatimagene Besadenovec (clinicaltrials.gov)
    P2, N=45, Recruiting, Candel Therapeutics, Inc. | N=30 --> 45
    Enrollment change
    |
    ProstAtak (aglatimagene besadenovec) • valacyclovir
    15d
    liMeliGhT: A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa (clinicaltrials.gov)
    P3, N=150, Active, not recruiting, Ocugen | Recruiting --> Active, not recruiting
    Enrollment closed
    17d
    PS-002 for the Treatment of IgA Nephropathy in Adults (clinicaltrials.gov)
    P1/2, N=32, Recruiting, Purespring Therapeutics Limited | Initiation date: Nov 2025 --> Feb 2026
    Trial initiation date • First-in-human
    17d
    GS-LHON-CLIN-08: A dose-ranging randomized, open-label study evaluating the effect of bilateral intravitreal injection of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy The REVISE Study (2025-523339-20-00)
    P1/2, N=14, Recruiting, Gensight Biologics
    New P1/2 trial
    17d
    Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency (clinicaltrials.gov)
    P3, N=32, Active, not recruiting, Ultragenyx Pharmaceutical Inc | Trial primary completion date: Feb 2025 --> Sep 2027
    Trial primary completion date