^
    4d
    The Preliminary Safety and Efficacy of RRG001 After Vitrectomy in Subjects With Proliferative Diabetic Retinopathy (PDR) (clinicaltrials.gov)
    P1, N=6, Recruiting, Li Xiaorong
    New P1 trial
    10d
    RestorAATion-2: A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD (clinicaltrials.gov)
    P1/2, N=24, Not yet recruiting, Wave Life Sciences Ltd.
    New P1/2 trial
    11d
    IVB102-101: Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis (clinicaltrials.gov)
    P1, N=18, Recruiting, InnoVec Biotherapeutics Inc. | Not yet recruiting --> Recruiting
    Enrollment open
    12d
    GenePHIT: Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (clinicaltrials.gov)
    P2, N=150, Recruiting, Asklepios Biopharmaceutical, Inc. | Trial completion date: Dec 2029 --> Dec 2030 | Trial primary completion date: Dec 2029 --> Dec 2030
    Trial completion date • Trial primary completion date • Gene therapy
    12d
    Serpentine-1: A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency (clinicaltrials.gov)
    P1, N=12, Recruiting, Krystal Biotech, Inc. | Trial completion date: Jun 2025 --> May 2026
    Trial completion date
    15d
    Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (clinicaltrials.gov)
    P1/2, N=85, Not yet recruiting, Essen Biotech
    New P1/2 trial • Gene therapy • Viral vector
    |
    busulfan
    15d
    Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 (clinicaltrials.gov)
    P1/2, N=34, Completed, MeiraGTx UK II Ltd | Active, not recruiting --> Completed | Trial completion date: Jan 2026 --> Apr 2024 | Trial primary completion date: Jan 2026 --> Apr 2024
    Trial completion • Trial completion date • Trial primary completion date • Gene therapy
    16d
    Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease (clinicaltrials.gov)
    P1/2, N=15, Recruiting, UniQure Biopharma B.V. | Trial completion date: Oct 2027 --> Oct 2029 | Trial primary completion date: Mar 2027 --> Mar 2029
    Trial completion date • Trial primary completion date
    |
    CHI3L1 (Chitinase 3-like 1) • GFAP (Glial Fibrillary Acidic Protein) • NEFL (Neurofilament Light Chain)
    17d
    Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A (clinicaltrials.gov)
    P1/2, N=3, Suspended, Nationwide Children's Hospital | Trial completion date: Jul 2024 --> Apr 2030 | Trial primary completion date: Mar 2024 --> Apr 2028
    Trial completion date • Trial primary completion date
    18d
    Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug (clinicaltrials.gov)
    P1/2, N=33, Recruiting, GeneCradle Inc
    New P1/2 trial • Gene therapy
    19d
    liMeliGhT: A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa (clinicaltrials.gov)
    P3, N=150, Recruiting, Ocugen
    New P3 trial • Gene therapy
    24d
    A Study to Evaluate Intravitreal JNJ-81201887 (AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) (clinicaltrials.gov)
    P2, N=300, Recruiting, Janssen Research & Development, LLC | Trial completion date: Jun 2026 --> Jan 2026
    Trial completion date
    25d
    BEYOND-9: A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (clinicaltrials.gov)
    P1/2, N=130, Not yet recruiting, Regeneron Pharmaceuticals
    New P1/2 trial
    26d
    NAN-CS101: NAN-101 in Patients With Class III Heart Failure (clinicaltrials.gov)
    P1, N=12, Recruiting, Asklepios Biopharmaceutical, Inc. | Trial completion date: Dec 2024 --> Dec 2026 | Trial primary completion date: Dec 2023 --> Dec 2026
    Trial completion date • Trial primary completion date
    26d
    A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants (clinicaltrials.gov)
    P=N/A, N=10, Recruiting, Shanghai BDgene Co., Ltd. | Trial completion date: Feb 2024 --> Feb 2025 | Trial primary completion date: Feb 2024 --> Feb 2025
    Trial completion date • Trial primary completion date • Gene therapy • Viral vector
    30d
    PDE6A Gene Therapy for Retinitis Pigmentosa (clinicaltrials.gov)
    P1/2, N=9, Active, not recruiting, STZ eyetrial | Trial primary completion date: Jul 2023 --> Jul 2027
    Trial primary completion date • Gene therapy
    1m
    Long-Term Follow-Up Study of AAV2hAQP1 for Radiation Induced Xerostomia (clinicaltrials.gov)
    P=N/A, N=22, Active, not recruiting, MeiraGTx, LLC | Enrolling by invitation --> Active, not recruiting | Trial completion date: Nov 2027 --> Mar 2027 | Trial primary completion date: Nov 2026 --> Mar 2027
    Enrollment closed • Trial completion date • Trial primary completion date
    1m
    AUDIOGENE: A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (clinicaltrials.gov)
    P1/2, N=12, Not yet recruiting, Sensorion
    New P1/2 trial
    1m
    RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD) (clinicaltrials.gov)
    P2, N=60, Completed, AbbVie | Active, not recruiting --> Completed
    Trial completion • Gene therapy
    1m
    Stargazer: A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (clinicaltrials.gov)
    P=N/A, N=120, Recruiting, bluebird bio | Not yet recruiting --> Recruiting
    Enrollment open
    1m
    Gene Therapy for Fanconi Anemia, Complementation Group A (clinicaltrials.gov)
    P2, N=5, Active, not recruiting, Rocket Pharmaceuticals Inc. | Recruiting --> Active, not recruiting | Trial completion date: Jun 2023 --> May 2026 | Trial primary completion date: Jun 2022 --> May 2026
    Enrollment closed • Trial completion date • Trial primary completion date • Gene therapy • Viral vector
    |
    CD34 (CD34 molecule)
    1m
    4D-310 in Adults With Fabry Disease and Cardiac Involvement (clinicaltrials.gov)
    P1/2, N=18, Active, not recruiting, 4D Molecular Therapeutics | Trial completion date: May 2028 --> Jan 2030 | Trial primary completion date: May 2024 --> Jan 2026
    Trial completion date • Trial primary completion date • Gene therapy
    1m
    An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (clinicaltrials.gov)
    P1/2, N=18, Active, not recruiting, 4D Molecular Therapeutics | Trial completion date: Jun 2027 --> Jun 2030 | Trial primary completion date: Jun 2023 --> Jan 2026
    Trial completion date • Trial primary completion date • Gene therapy
    1m
    A Long-term Follow-up Study to Evaluate SKG0106 in the Treatment of Patients With nAMD (clinicaltrials.gov)
    P=N/A, N=83, Recruiting, Skyline Therapeutics
    New trial
    2ms
    RIDGE-1: Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (clinicaltrials.gov)
    P1, N=15, Recruiting, Tenaya Therapeutics | Not yet recruiting --> Recruiting
    Enrollment open
    2ms
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=6, Recruiting, Solid Biosciences Inc. | Not yet recruiting --> Recruiting
    Enrollment open • Gene therapy
    2ms
    AAV Gene Therapy Clinical Study in Adult Classic PKU (clinicaltrials.gov)
    P1/2, N=12, Not yet recruiting, NGGT (Suzhou) Biotechnology Co., Ltd.
    New P1/2 trial • Gene therapy
    2ms
    A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa (clinicaltrials.gov)
    P3, N=4, Recruiting, Janssen Pharmaceutical K.K. | Trial completion date: May 2029 --> Oct 2029
    Trial completion date
    2ms
    Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE] (clinicaltrials.gov)
    P2, N=27, Completed, Nanoscope Therapeutics Inc. | Active, not recruiting --> Completed
    Trial completion
    2ms
    Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (clinicaltrials.gov)
    P1/2, N=15, Active, not recruiting, Prevail Therapeutics | Trial completion date: Dec 2028 --> May 2028 | Trial primary completion date: Dec 2028 --> May 2028
    Trial completion date • Trial primary completion date
    |
    prednisone • sirolimus
    2ms
    Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients (clinicaltrials.gov)
    P=N/A, N=25, Not yet recruiting, Forge Biologics, Inc
    New trial • Gene therapy
    2ms
    Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) (clinicaltrials.gov)
    P1/2, N=40, Recruiting, National Institute of Allergy and Infectious Diseases (NIAID) | Trial completion date: Dec 2024 --> Dec 2032 | Trial primary completion date: Dec 2024 --> Dec 2032
    Trial completion date • Trial primary completion date
    |
    busulfan • Kepivance (palifermin)
    2ms
    Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy (clinicaltrials.gov)
    P1, N=6, Active, not recruiting, Xiamen Ophthalmology Center Affiliated to Xiamen University
    New P1 trial
    2ms
    Gene Therapy for ACM Due to a PKP2 Pathogenic Variant (clinicaltrials.gov)
    P1/2, N=10, Recruiting, Lexeo Therapeutics | Not yet recruiting --> Recruiting
    Enrollment open • Gene therapy
    2ms
    Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1) (clinicaltrials.gov)
    P1/2, N=9, Active, not recruiting, West China Hospital
    New P1/2 trial • Gene therapy
    2ms
    Phase 1b Safety and Preliminary Efficacy of Bilateral Intraputaminal Delivery of AAV2 GDNF (AB-1005) in Participants With Mild or Moderate Parkinson's Disease (AAN 2024)
    Analysis of this phase 1b trial is ongoing; topline 18-month data will be presented. These preliminary findings suggest AAV2-GDNF is well tolerated in participants with PD, demonstrating general stability of the Mild Cohort and possible clinical benefit in the Moderate Cohort. A phase 2 randomized controlled study is planned to confirm these findings.
    Clinical • P1 data
    2ms
    Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1 (clinicaltrials.gov)
    P1, N=52, Recruiting, Arrowhead Pharmaceuticals | Not yet recruiting --> Recruiting
    Enrollment open
    2ms
    ATMOSPHERE: Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD (clinicaltrials.gov)
    P2/3, N=300, Recruiting, AbbVie | Phase classification: P2b --> P2/3
    Phase classification • Gene therapy
    |
    Lucentis (ranibizumab)
    3ms
    Study of UX701 Gene Transfer for the Treatment of Wilson Disease (clinicaltrials.gov)
    P1/2, N=78, Active, not recruiting, Ultragenyx Pharmaceutical Inc | Trial completion date: Jan 2031 --> Nov 2031 | Trial primary completion date: Jan 2026 --> Aug 2026
    Trial completion date • Trial primary completion date
    3ms
    LTFU: Long-Term Follow-up Study for Patients From AVXS-101-CL-101 (clinicaltrials.gov)
    P=N/A, N=13, Active, not recruiting, Novartis Gene Therapies | Trial completion date: Dec 2033 --> Dec 2030 | Trial primary completion date: Dec 2033 --> Dec 2030
    Trial completion date • Trial primary completion date
    3ms
    A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD) (clinicaltrials.gov)
    P2, N=87, Not yet recruiting, Asklepios Biopharmaceutical, Inc.
    New P2 trial
    3ms
    A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel (clinicaltrials.gov)
    P=N/A, N=150, Recruiting, bluebird bio
    New trial