ANB-002 / Biocad - LARVOL DELTA

An open-label, non-controlled study to evaluate the safety, pharmacodynamics, and efficacy of single-dose administration of ANB-002 in patients with hemophilia B. (ChiCTR) - P=N/A | N=8 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.; Institute of Hematolog

New trial • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

ARVENACOGENE SANPARVOVEC, A NOVEL OPTION FOR HEMOPHILIA B GENE THERAPY: FIRST-IN-HUMAN FINDINGS (EHA 2025) - P1/2 | "ANB-002, a new promising gene therapy option for hemophilia B, was well tolerated when administered once at the dose of 2.5×1012 vg/kg or 5×1012 vg/kg. In six patients, FIX activity exceeded 5% and remained at this level throughout the 52-week period. Four patients with stable activity had no bleeding and no need for exogenous FIX prophylaxis."

Gene therapy • P1 data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

MAGNOLIA: An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B (clinicaltrials.gov) - P3 | N=24 | Recruiting | Sponsor: Biocad | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN) (clinicaltrials.gov) - P1/2 | N=28 | Recruiting | Sponsor: Biocad | N=12 ➔ 28

Enrollment change • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

MAGNOLIA: An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients with Hemophilia B (clinicaltrials.gov) - P3 | N=24 | Not yet recruiting | Sponsor: Biocad

New P3 trial • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases