BBM-H803 / Belief Biomed - LARVOL DELTA

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs (clinicaltrials.gov) - P2/3 | N=55 | Recruiting | Sponsor: Shanghai Xinzhi BioMed Co., Ltd. | Phase classification: P1/2 ➔ P2/3 | N=12 ➔ 55 | Trial completion date: Jun 2030 ➔ May 2031 | Trial primary completion date: Jun 2026 ➔ May 2027

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Prophylaxis CTLA4-ig reduces anti-AAV neutralizing antibodies in AAV gene therapy for hemophilia (ASH 2025) - P=N/A, P1 | "Abatacept(CTLA4-Ig, 5 mg/kg) or an equivalent PBS was administered intraperitoneally 3 days prior to vectorinfusion, followed by twice-weekly maintenance dosing for 6 weeks...Prophylaxis CTLA4-Ig (5 mg/kg) was administered3 days prior to AAV vector (BBM-H803) infusion... Prophylaxis administration of CTLA4-Ig effectively suppresses anti-AAV Nab formationfollowing AAV gene therapy in hemophilia A patients, creating a potential immunological tolerancewindow for re-treatment."

Gene therapy • IO biomarker • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases • CD86

A Study of FVIII Gene Therapy for Hemophilia A (clinicaltrials.gov) - P1 | N=8 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Not yet recruiting ➔ Recruiting | Trial primary completion date: Sep 2023 ➔ Dec 2024

Enrollment open • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases