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    DRUG CLASS:

    Gene transference

    Related drugs:
    1d
    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
    6d
    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
    8d
    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)
    9d
    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
    14d
    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
    15d
    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
    15d
    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)
    16d
    RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) (clinicaltrials.gov)
    P3, N=2, Not yet recruiting, REGENXBIO Inc.
    New P3 trial
    16d
    ARTEMIS: Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (clinicaltrials.gov)
    P3, N=284, Recruiting, Adverum Biotechnologies, Inc. | Trial completion date: Mar 2030 --> Nov 2030 | Trial primary completion date: Apr 2027 --> Dec 2026
    Trial completion date • Trial primary completion date
    20d
    AAVIATE: RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD) (clinicaltrials.gov)
    P2, N=140, Recruiting, AbbVie | Trial completion date: Aug 2026 --> Feb 2027 | Trial primary completion date: Oct 2025 --> Aug 2026
    Trial completion date • Trial primary completion date
    |
    Lucentis (ranibizumab)
    22d
    LX2020-01: Gene Therapy for ACM Due to a PKP2 Pathogenic Variant (clinicaltrials.gov)
    P1/2, N=10, Active, not recruiting, Lexeo Therapeutics | Recruiting --> Active, not recruiting | Trial primary completion date: Feb 2026 --> Dec 2026
    Enrollment closed • Trial primary completion date
    23d
    GRIT-PKP2: Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant (clinicaltrials.gov)
    P=N/A, N=10, Enrolling by invitation, Lexeo Therapeutics | Not yet recruiting --> Enrolling by invitation
    Enrollment open