^
    3d
    A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov)
    P1/2, N=60, Recruiting, Solid Biosciences Inc. | N=40 --> 60
    Enrollment change
    4d
    FALCON: A Study of SGT-212 Gene Therapy in Friedreich's Ataxia (clinicaltrials.gov)
    P1, N=10, Recruiting, Solid Biosciences Inc. | Not yet recruiting --> Recruiting
    Enrollment open • First-in-human
    4d
    A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) (clinicaltrials.gov)
    P1/2, N=15, Recruiting, Prevail Therapeutics | Trial completion date: Oct 2030 --> Nov 2031 | Trial primary completion date: Oct 2030 --> Nov 2031
    Trial completion date • Trial primary completion date
    4d
    AXIS: First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration (clinicaltrials.gov)
    P1, N=12, Not yet recruiting, Axovia Therapeutics
    New P1 trial • First-in-human
    8d
    Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC). (clinicaltrials.gov)
    P3, N=15, Recruiting, Lantu Biopharma
    New P3 trial • Real-world evidence
    9d
    GenePHIT: Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (clinicaltrials.gov)
    P2, N=150, Active, not recruiting, AskBio Inc | Recruiting --> Active, not recruiting
    Enrollment closed
    14d
    PS-002 for the Treatment of IgA Nephropathy in Adults (clinicaltrials.gov)
    P1/2, N=32, Recruiting, Purespring Therapeutics Limited | Not yet recruiting --> Recruiting
    Enrollment open
    16d
    Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer: Current Evidence and Future Directions. (PubMed, Cancers (Basel))
    Nadofaragene firadenovec, a recombinant adenovirus delivering interferon alpha-2b (IFNα2b), is the first FDA-approved gene therapy for BCG-unresponsive NMIBC with carcinoma in situ (CIS)...Cretostimogene grenadenorepvec (CG0070), an oncolytic vector, demonstrated a 47% 6-month CR rate in a phase II study (NCT02365818). Detalimogene voraplasmid (EG-70), a nonviral gene therapy, demonstrated a 47% 6-month CR in a phase I/II study (NCT04752722). Future advances are likely to focus on patient selection, novel vectors, and combination strategies to improve treatment outcomes. Gene therapy represents a significant addition to the bladder cancer treatment landscape by offering bladder-sparing alternatives where conventional therapies are limited.
    Review • Journal
    |
    IFNA1 (Interferon Alpha 1)
    |
    Adstiladrin (nadofaragene firadenovec-vncg) • cretostimogene grenadenorepvec (CG0070) • detalimogene voraplasmid (EG-70)
    17d
    RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) (clinicaltrials.gov)
    P3, N=2, Recruiting, REGENXBIO Inc. | Not yet recruiting --> Recruiting
    Enrollment open
    22d
    A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) (clinicaltrials.gov)
    P3, N=15, Recruiting, Taysha Gene Therapies, Inc. | Phase classification: P1/2 --> P3 | Trial completion date: Jan 2032 --> Jun 2031 | Trial primary completion date: Nov 2028 --> Jun 2031
    Phase classification • Trial completion date • Trial primary completion date
    23d
    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, Recruiting, Prevail Therapeutics | N=20 --> 32
    Enrollment change
    |
    sirolimus
    23d
    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 | Not yet recruiting --> Recruiting
    Enrollment open • Adverse events • Real-world evidence
    |
    Lucentis (ranibizumab)