^
    21h
    Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (clinicaltrials.gov)
    P1, N=12, Active, not recruiting, Elixirgen Therapeutics, Inc. | Suspended --> Active, not recruiting
    Enrollment closed
    1d
    Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE) (clinicaltrials.gov)
    P1/2, N=6, Completed, Assistance Publique - Hôpitaux de Paris | Active, not recruiting --> Completed
    Trial completion
    2d
    ARMOR: A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS) (clinicaltrials.gov)
    P1, N=23, Recruiting, Insmed Gene Therapy LLC | Not yet recruiting --> Recruiting
    Enrollment open
    |
    SOD1 (Superoxide Dismutase 1)
    3d
    AGT4HB: Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B (clinicaltrials.gov)
    P1, N=14, Active, not recruiting, St. Jude Children's Research Hospital | Trial completion date: Dec 2025 --> Mar 2026 | Trial primary completion date: Dec 2025 --> Mar 2026
    Trial completion date • Trial primary completion date
    7d
    An Exploratory Clinical Study of JV001 in the Treatment of Patients With Heart Failure Due to Dilated Cardiomyopathy (ChiCTR2600117671)
    P=N/A, N=12, Completed, Shanghai East Hospital; Shanghai East Hospital
    New trial
    7d
    A Phase III, Randomized, Open-Label, Active-Controlled Study to Evaluate the Efficacy and Safety of Subretinal Injection of LX102 in Participants with Neovascular Age-Related Macular Degeneration – The STELLAR Trial (ChiCTR2500115643)
    P3, N=332, Recruiting, Shanghai General Hospital; Shanghai Langsheng Biotechnology Co., Ltd
    New P3 trial
    |
    Eylea (aflibercept intravitreal)
    7d
    A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS) (ChiCTR2600117343)
    P1/2, N=21, Not yet recruiting, Peking University Third Hospital; Peking University Third Hospital
    New P1/2 trial
    9d
    ATA-200 Gene Therapy Trial in Patients With LGMDR5 (clinicaltrials.gov)
    P1, N=4, Active, not recruiting, Atamyo Therapeutics | Recruiting --> Active, not recruiting | Phase classification: P1/2 --> P1
    Enrollment closed • Phase classification
    10d
    Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (clinicaltrials.gov)
    P1, N=12, Suspended, Elixirgen Therapeutics, Inc. | Recruiting --> Suspended
    Trial suspension
    15d
    Safety and Efficacy Evaluation of LX111 Gene Therapy in DME Patients (clinicaltrials.gov)
    P1, N=32, Not yet recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
    New P1 trial
    17d
    ASPIRE-FTD: A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN) (clinicaltrials.gov)
    P1/2, N=9, Recruiting, AviadoBio Ltd | Trial completion date: Oct 2030 --> Mar 2030
    Trial completion date
    |
    GRN (Granulin Precursor)
    17d
    Long-term Follow-up Gene Therapy Study for RPGR- XLRP (clinicaltrials.gov)
    P=N/A, N=42, Completed, Janssen Research & Development, LLC | Active, not recruiting --> Completed | Trial completion date: Nov 2026 --> Sep 2025 | Trial primary completion date: Nov 2026 --> Sep 2025
    Trial completion • Trial completion date • Trial primary completion date