^
    3d
    Expanded Access to RGX-202 (clinicaltrials.gov)
    P=N/A, N=0, Temporarily Not Available, REGENXBIO Inc.
    New trial
    5d
    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
    5d
    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
    9d
    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
    9d
    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
    15d
    Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration (clinicaltrials.gov)
    P2, N=15, Recruiting, Adverum Biotechnologies, Inc.
    New P2 trial
    16d
    CLTW888A11301: Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy (clinicaltrials.gov)
    P3, N=4, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed
    Trial completion
    16d
    RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) (clinicaltrials.gov)
    P3, N=2, Active, not recruiting, REGENXBIO Inc. | Suspended --> Active, not recruiting
    Enrollment closed
    17d
    Acclaim-2: Quaratusugene Ozeplasmid (Reqorsa) in Combination With Pembrolizumab in Previously Treated Non-Small Lung Cancer (clinicaltrials.gov)
    P1, N=5, Terminated, Genprex, Inc. | Phase classification: P1/2 --> P1
    Phase classification
    |
    EGFR (Epidermal growth factor receptor) • PD-L1 (Programmed death ligand 1) • ALK (Anaplastic lymphoma kinase)
    |
    ALK mutation
    |
    Keytruda (pembrolizumab) • docetaxel • Cyramza (ramucirumab) • Reqorsa (quaratusugene ozeplasmid)
    17d
    Acclaim-3: Quaratusugene Ozeplasmid (Reqorsa) and Atezolizumab Maintenance Therapy in ES-SCLC Patients (clinicaltrials.gov)
    P1/2, N=62, Recruiting, Genprex, Inc. | Trial primary completion date: Feb 2026 --> Jun 2026
    Trial primary completion date
    |
    Tecentriq (atezolizumab) • Reqorsa (quaratusugene ozeplasmid)
    24d
    SGT-003-301: A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (2025-522949-22-00)
    P2/3, N=20, Not yet recruiting, Solid Biosciences Inc.
    New P2/3 trial
    29d
    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, Recruiting, Taysha Gene Therapies, Inc. | Not yet recruiting --> Recruiting
    Enrollment open