Rare Daily Staff
China’s National Medical Products Administration has cleared the Investigational New Drug application for Belief Biomed’s hemophilia B gene therapy BBM-H901, the first IND approval for an intravenous infusion gene therapy for rare diseases in China.
The company said the IND approval of BBM-H901 is a significant milestone for the company and opens a “new chapter” for the story of gene therapy in China. Belief expects to begin a phase 1/2 clinical study shortly.
“Gene therapy is the third wave of biopharma industry revolution, following small molecules and antibodies. The gene therapy market has proliferated in recent years. Novartis, Johnson & Johnson, and other pharmaceutical giants have all waged bets in the field,” said Jane Zheng, CEO of Belief Biomed. “With our robust R&D team and state-of-art technologies, Belief Biomed has become one of the few startups that can rival big pharmas in Europe and the United States.”
Hemophilia B is a rare bleeding disorder caused by mutations in the F9 gene resulting in insufficient levels of the blood clotting protein factor IX. Symptoms can range from mild to severe depending on the level of factor IX in the blood and can range from prolonged bleeding after injury to frequent spontaneous bleeds into the muscles and joints and organs in severe cases that cause pain and other problems. Bleeding in the brain can lead to death if untreated.
BBM-H901 is a cutting-edge bio-engineered adeno-associated viral (AAV) vector utilizing a novel recombinant AAV capsid, containing a codon-optimized human factor IX gene under the control of a liver-specific promoter. Belief owns the proprietary patents of the capsid and the cassette. The company said BBM-H901 has demonstrated high efficacy and safety with the factor IX well-expressed in patient’s plasma. Belief said it expects BBM-H901 can eliminate bleeding and liberate patients from regular treatment.
An investigator-initiated trial of BBM-H901 is one of the earliest clinical trials for AAV Gene therapy carried out in China. It started in 2019, and the clinical data demonstrated excellent safety and efficacy. The annualized bleeding rate declined significantly after administration. All patients had stable FIX activity during the follow-up with no indication of serious adverse events.
Belief has received more than $150 million of investment in venture funding. It maintains facilities in North Carolina, Shanghai, Hong Kong and Beijing. Xiao Xiao, who currently serves as the president and chief scientific officer of the company, founded the company.
Xiao has more than 35-years of experience in engineering AAV. His work has been developed into the first gene therapy clinical trial for DMD in the United States at Bamboo Therapeutics, which Pfizer acquired for more than $650 million. Prior to that, Xiao co-founded AskBio, which Bayer acquired for more than $4 billion.
Photo: Xiao Xiao, founder and chief scientific officer of Belief Biomed