^
    2ms
    CHORD: A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations (clinicaltrials.gov)
    P1/2, N=36, Recruiting, Regeneron Pharmaceuticals | Trial primary completion date: Feb 2032 --> Feb 2029
    Trial primary completion date
    2ms
    AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov)
    P2/3, N=65, Recruiting, REGENXBIO Inc. | Trial primary completion date: Feb 2026 --> Aug 2026
    Trial primary completion date
    2ms
    Landscape: A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa (clinicaltrials.gov)
    P2, N=10, Active, not recruiting, Beacon Therapeutics | Trial primary completion date: Jun 2027 --> Dec 2029
    Trial primary completion date
    2ms
    OTL-200-11: OTL-200 Long Term Insertional Oncogenesis Study (2025-522275-28-00)
    P4, N=5, Not yet recruiting, Orchard Therapeutics (Europe) Limited
    New P4 trial
    2ms
    AAV9/AP4M1-DK01: A Phase IIb, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50) (2025-524481-15-00)
    P1/2, N=2, Not yet recruiting, Rigshospitalet, Elpida Therapeutics SPC
    New P1/2 trial
    2ms
    Landscape: A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis Pigmentosa (clinicaltrials.gov)
    P2, N=10, Active, not recruiting, Beacon Therapeutics | Recruiting --> Active, not recruiting
    Enrollment closed
    2ms
    LuTK03: Aglatimagene Besadenovec + Prodrug and Pembrolizumab vs Docetaxel for Stage IV Non-Squamous NSCLC Progressing on Pembrolizumab (AURORA) (clinicaltrials.gov)
    P3, N=500, Recruiting, Candel Therapeutics, Inc.
    New P3 trial
    |
    Keytruda (pembrolizumab) • docetaxel • ProstAtak (aglatimagene besadenovec) • valacyclovir
    2ms
    Expanded Access to RGX-202 (clinicaltrials.gov)
    P=N/A, N=0, Temporarily Not Available, REGENXBIO Inc.
    New trial
    2ms
    An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia (clinicaltrials.gov)
    P1/2, N=6, Active, not recruiting, Lantu Biopharma | Recruiting --> Active, not recruiting
    Enrollment closed
    2ms
    An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy (clinicaltrials.gov)
    P1/2, N=20, Active, not recruiting, Lantu Biopharma | Recruiting --> Active, not recruiting
    Enrollment closed
    2ms
    CCPK850X2202: A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis Pigmentosa (clinicaltrials.gov)
    P1/2, N=12, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed
    Trial completion • First-in-human
    2ms
    Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH (clinicaltrials.gov)
    P2/3, N=36, Recruiting, Ultragenyx Pharmaceutical Inc | Trial completion date: Jul 2027 --> Mar 2029 | Trial primary completion date: Jul 2027 --> Mar 2029
    Trial completion date • Trial primary completion date