dalnacogene ponparvovec (BBM-H901) / Belief Biomed, Takeda - LARVOL DELTA

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug (clinicaltrials.gov) - P2/3 | N=32 | Active, not recruiting | Sponsor: Shanghai Belief-Delivery BioMed Co., Ltd | Phase classification: P3 ➔ P2/3

Phase classification • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Gene transfer with BBM-H901: results of long term follow up in Chinese Hemophilia B patients (ISTH 2025) - P=N/A | "All the nine participants had no bleeding events and replacement therapy in long follow up period. Table or Figure Upload"

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

The Cost-effectiveness Analysis of Gene Therapy for Severe Hemophilia B Patients in China: A Microsimulation Experiment Study (ISPOR 2025) - "BBM-H901 was shown to be an economically dominant alternative, offering lower costs and higher QALYs, compared to SHL rFIX for hemophilia B patients in China."

Clinical • Cost effectiveness • Gene therapy • HEOR • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Orthopedics • Rare Diseases

Belief BioMed and Takeda China jointly announced BBM-H901 (Dalnacogene Ponparvovec Injection), China's first hemophilia B gene therapy, was officially approved (PRNewswire) - "Belief BioMed...and Takeda China today jointly announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by the National Medical Products Administration (NMPA) for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency). As the first approved hemophilia B gene therapy in China, BBM-H901 is developed and manufactured by BBM, and Takeda China is responsible for its commercialization in mainland China, Hong Kong and Macau."

China approval • Hemophilia B

Gene Therapy for Chinese Hemophilia B (clinicaltrials.gov) - P=N/A | N=10 | Completed | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Active, not recruiting ➔ Completed

Trial completion • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Efficacy and Safety of an Engineered, Liver-Tropic Adeno-Associated Virus Vector Expressing Factor IX Padua Administered with Prophylactic Glucocorticoids in Patients with Hemophilia B: A Multi-Center, Single-Arm, Phase 3 Trial (ASH 2024) - P3 | "Eligible participants were intravenously infused with a single dose of 5 × 10¹² vector genomes (vg)/kg of BBM-H901 after 1 day of prophylactic oral prednisone at 1 mg/kg/d. Besides, the trial also supports the safety and tolerability. In conclusion, BBM-H901 gene therapy is superior to prophylactic treatment with respect to the ABR, and it had a favorable safety profile."

Clinical • P3 data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Sustained efficacy of gene transfer with BBM-H901 in Chinese Hemophilia B patients: results of long term follow up (ISTH 2024) - "Ten patients were follow-up for a median 130 weeks (Range 130 to 209 weeks). At all patients’ latest follow-up, the mean(±SD) FIX:C level was 38.36(±19.89)% measured by one stage APTT based method using Dade Actin FSL（Siemens， Germany） reagents. The mean FIX:C level at each time points maintain stable expression after BBM-H901 treatment (Figure)."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Gene Therapy for Chinese Hemophilia B (clinicaltrials.gov) - P=N/A | N=10 | Active, not recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug (clinicaltrials.gov) - P3 | N=32 | Active, not recruiting | Sponsor: Shanghai Belief-Delivery BioMed Co., Ltd | Recruiting ➔ Active, not recruiting | Phase classification: P1/2 ➔ P3 | N=12 ➔ 32

Enrollment change • Enrollment closed • Gene therapy • Phase classification • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old (clinicaltrials.gov) - P1 | N=9 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital

Gene therapy • New P1 trial • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Gene Therapy for Chinese Hemophilia B (clinicaltrials.gov) - P=N/A | N=10 | Active, not recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital | Trial completion date: Dec 2022 ➔ Dec 2039 | Trial primary completion date: Feb 2022 ➔ Dec 2039

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

Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. (PubMed, Lancet Haematol) - P=N/A | "This pilot study suggests that liver-tropic BBM-H901 is safe 1 year after infusion. Vector derived FIX:C concentration is sufficiently high to prevent bleeding events and minimise the need for replacement therapy in small populations with haemophilia B. These findings support further study."

Journal • P1 data • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug (clinicaltrials.gov) - P1/2; N=12; Recruiting; Sponsor: Shanghai Belief-Delivery BioMed Co., Ltd

New P1/2 trial • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Belief BioMed Completed Dosing of the First Subject in the Registrational Gene Therapy Clinical Trial for Hemophilia B (PRNewswire) - "On December 30th, 2021, Belief BioMed Group (BBM) announced that it has successfully dosed the first subject in the registrational gene therapy clinical trial by intravenous (IV) infusion of BBM-H901, an adeno-associated virus (AAV) vector expressing factor IX gene for treatment of adult male hemophilia B patients....This registrational clinical study (CTR20212816) is a multi-center, single-arm, open-label and single-treatment study. Its objective is to evaluate the safety, tolerability, pharmacokinetics, as well as long-term efficacy and safety of a single intravenous infusion of BBM-H901 in hemophilia B patients ≥18 years of age with endogenous blood coagulation factor IX (FIX) activity ≤2 IU/dL (≤2%)."

Trial status • Genetic Disorders • Hemophilia

Gene Therapy for Chinese Hemophilia B (clinicaltrials.gov) - P=N/A; N=10; Active, not recruiting; Sponsor: Institute of Hematology & Blood Diseases Hospital; Recruiting ➔ Active, not recruiting; Trial completion date: Nov 2021 ➔ Dec 2022; Trial primary completion date: Nov 2021 ➔ Feb 2022

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

Belief Biomed’s Gene Therapy for Hemophilia B Receives NMPA IND Approval: First Intravenous Infusion Gene Therapy for Rare Disease in China (PRNewswire) - P=NA, N=9; NCT04135300; "On August 10th, 2021, Belief Biomed announced that China's National Medical Products Administration (NMPA) has cleared the IND (Investigational New Drug) application for Belief Biomed's BBM-H901....The company will advance Phase 1/2 clinical study shortly....The Investigator Initiated Trial (IIT) of BBM-H901 is one of the earliest clinical trials for AAV Gene Therapy carried out in China. The IIT of BBM-H901 started in 2019, and the clinical data demonstrated excellent safety and efficacy. The annualized bleeding rate (ABR) declined significantly after administration. All patients had stable FIX activity during the follow-up with no indication of SAE (Serious Adverse Event)."

Clinical data • New P1/2 trial • Genetic Disorders • Hemophilia