^
    3d
    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
    3d
    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, Active, not recruiting, Wave Life Sciences Ltd. | Recruiting --> Active, not recruiting
    Enrollment closed
    6d
    Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS) (clinicaltrials.gov)
    P3, N=284, Recruiting, Adverum Biotechnologies, Inc.
    New P3 trial
    |
    Eylea (aflibercept intravitreal)
    6d
    ASPIRE: Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome (clinicaltrials.gov)
    P3, N=3, Not yet recruiting, Taysha Gene Therapies, Inc.
    New P3 trial
    9d
    A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov)
    P2, N=10, Completed, Orchard Therapeutics | Active, not recruiting --> Completed
    Trial completion
    |
    CD14 (CD14 Molecule)
    11d
    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=305, Completed, Janssen Research & Development, LLC | Active, not recruiting --> Completed | Trial completion date: Dec 2026 --> Feb 2026
    Trial completion • Trial completion date
    |
    prednisone
    11d
    Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov)
    P1/2, N=32, Active, not recruiting, Prevail Therapeutics | Recruiting --> Active, not recruiting
    Enrollment closed • First-in-human
    |
    sirolimus
    12d
    RGX-111 Gene Therapy in Patients With MPS I (clinicaltrials.gov)
    P1/2, N=21, Suspended, REGENXBIO Inc. | Trial completion date: Oct 2024 --> Mar 2029 | Active, not recruiting --> Suspended | Trial primary completion date: Mar 2023 --> Jul 2027
    Trial completion date • Trial suspension • Trial primary completion date • First-in-human
    14d
    A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) (clinicaltrials.gov)
    P3, N=49, Completed, Ultragenyx Pharmaceutical Inc | Active, not recruiting --> Completed
    Trial completion
    |
    prednisolone
    14d
    A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) (clinicaltrials.gov)
    P1/2, N=15, Recruiting, Prevail Therapeutics | Active, not recruiting --> Recruiting
    Enrollment open
    |
    prednisone • sirolimus • methylprednisolone sodium succinate
    15d
    Safety and Efficacy Evaluation of LX111 Gene Therapy in DME Patients (clinicaltrials.gov)
    P1, N=32, Recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Not yet recruiting --> Recruiting
    Enrollment open
    18d
    Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations (clinicaltrials.gov)
    P1, N=9, Active, not recruiting, Innostellar Biotherapeutics Co.,Ltd | Phase classification: P1/2 --> P1
    Phase classification