^
    1d
    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
    1d
    PRIME-0101: A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD ) (clinicaltrials.gov)
    P1/2, N=12, Enrolling by invitation, Prime Medicine, Inc. | Active, not recruiting --> Enrolling by invitation
    Enrollment open
    4d
    A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency (clinicaltrials.gov)
    P3, N=13, Active, not recruiting, Shanghai Vitalgen BioPharma Co., Ltd. | Not yet recruiting --> Active, not recruiting | Phase classification: P1 --> P3
    Enrollment closed • Phase classification
    5d
    AGT4HB: Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B (clinicaltrials.gov)
    P1, N=14, Completed, St. Jude Children's Research Hospital | Active, not recruiting --> Completed
    Trial completion
    6d
    VTx-002-01-001: A Phase 1/2 Study of the Safety and Tolerability of ICM VTx-002 in participants with ALS (2025-522697-37-00)
    P1/2, N=2, Not yet recruiting, VectorY Therapeutics B.V.
    New P1/2 trial
    |
    FUS (FUS RNA Binding Protein)
    7d
    VGN-R09b-101: A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Patients With Parkinson's Disease (clinicaltrials.gov)
    P1/2, N=39, Active, not recruiting, Shanghai Vitalgen BioPharma Co., Ltd. | Not yet recruiting --> Active, not recruiting | Trial completion date: Jul 2031 --> Sep 2030 | Trial primary completion date: Jul 2027 --> Sep 2026
    Enrollment closed • Trial completion date • Trial primary completion date
    7d
    Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients (clinicaltrials.gov)
    P1, N=2, Completed, Zhongmou Therapeutics | Recruiting --> Completed | Trial completion date: Oct 2027 --> Jan 2026
    Trial completion • Trial completion date
    9d
    OTL-103-4: A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome (clinicaltrials.gov)
    P3, N=10, Recruiting, Fondazione Telethon | Not yet recruiting --> Recruiting
    Enrollment open
    |
    Rituxan (rituximab) • fludarabine IV • busulfan • Granocyte (lenograstim) • plerixafor
    10d
    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 | Trial completion date: Apr 2034 --> Jun 2032
    Trial completion date • Real-world evidence
    11d
    Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD) (clinicaltrials.gov)
    P3, N=561, Recruiting, AbbVie
    Trial initiation date • Adverse events • Real-world evidence
    |
    Lucentis (ranibizumab)
    14d
    A Study to Assess Safety and Tolerability of PCRX-201 in Subjects With Painful Osteoarthritis of the Knee (clinicaltrials.gov)
    P2, N=135, Active, not recruiting, Pacira Pharmaceuticals, Inc | Recruiting --> Active, not recruiting | Trial completion date: Mar 2032 --> Jul 2032 | Trial primary completion date: Mar 2027 --> Jul 2032
    Enrollment closed • Trial completion date • Trial primary completion date
    |
    CXCL8 (Chemokine (C-X-C motif) ligand 8)
    16d
    NSR-CHM-OS2 273CH201: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1)nand in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study (2023-507994-16-00)
    P2/3, N=54, Recruiting, Biogen Idec Research Limited | Not yet recruiting --> Recruiting
    Enrollment open