^
    3d
    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)
    8d
    CHORD: A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations (clinicaltrials.gov)
    P1/2, N=30, Recruiting, Regeneron Pharmaceuticals | Trial completion date: Apr 2031 --> Feb 2032 | Trial primary completion date: Apr 2031 --> Feb 2032
    Trial completion date • Trial primary completion date
    10d
    LTF-304: Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product (clinicaltrials.gov)
    P2/3, N=64, Active, not recruiting, bluebird bio | Phase classification: P --> P2/3
    Phase classification
    13d
    LIGHTHOUSE: ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (clinicaltrials.gov)
    P3, N=97, Recruiting, Atsena Therapeutics Inc. | Phase classification: P1/2 --> P3 | N=21 --> 97 | Trial completion date: Oct 2029 --> Apr 2033 | Trial primary completion date: Oct 2025 --> Apr 2028
    Phase classification • Enrollment change • Trial completion date • Trial primary completion date
    13d
    Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy (clinicaltrials.gov)
    P1, N=14, Completed, National Institute of Neurological Disorders and Stroke (NINDS) | Active, not recruiting --> Completed | Trial completion date: Apr 2035 --> Apr 2026 | Trial primary completion date: Jun 2030 --> Apr 2026
    Trial completion • Trial completion date • Trial primary completion date • First-in-human
    15d
    BrAAVe: A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 (clinicaltrials.gov)
    P1/2, N=32, Recruiting, Sanofi | Trial completion date: Feb 2029 --> Apr 2032
    Trial completion date
    16d
    ATMOSPHERE: Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD (clinicaltrials.gov)
    P2/3, N=671, Active, not recruiting, AbbVie | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    Lucentis (ranibizumab)
    19d
    ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene (clinicaltrials.gov)
    P1, N=10, Active, not recruiting, San Rocco Therapeutics | Trial completion date: Jul 2025 --> Jul 2027 | Trial primary completion date: Jul 2025 --> Jul 2027
    Trial completion date • Trial primary completion date
    |
    busulfan • Neupogen (filgrastim)
    20d
    To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A (clinicaltrials.gov)
    P1/2, N=7, Active, not recruiting, Gritgen Therapeutics Co., Ltd.
    New P1/2 trial
    22d
    Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN) (clinicaltrials.gov)
    P1, N=4, Recruiting, University of Texas Southwestern Medical Center
    New P1 trial
    22d
    GALILEO-3: A Gaucher Disease Gene Therapy Trial With FLT201 (clinicaltrials.gov)
    P3, N=45, Recruiting, Spur Therapeutics | Not yet recruiting --> Recruiting
    Enrollment open
    22d
    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