Belief Biomed's Gene Therapy for Hemophilia B Receives NMPA IND Approval: First Intravenous Infusion Gene Therapy for Rare Disease in China

SHANGHAI,Aug. 10,2021 --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. This marks the first IND approval for Intravenous (i.v.) infusion gene therapy for Hemophilia B in China,and the first Intravenous infusion gene therapy for rare diseases in China. The company will advance 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 Dr 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."

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 Biomed owns the proprietary patents of the capsid and the cassette. Leveraging the serum-free production and chromatographic purification process independently developed by Belief Biomed,BBM-H901 has demonstrated high efficacy and safety with the FIX (Factor IX) well-expressed in patient's plasma,achieving "one-time treatment,long-term benefit" effect. BBM expect BBM-H901 can eliminate bleeding and liberate patients from regular treatment.

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(NCT04135300) 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).

Belief Biomed submitted its original IND application to NMPA in April 2021 and received clearance on August 6th,2021.

About Belief Biomed

Founded in 2016,Belief BioMed (BBM) is dedicated to developing and commercializing AAV-based gene therapies. The company has received over US$ 150 million of investment from prestigious VC firms including Qiming Venture Partners,Lilly Asia Ventures,Sherpa Healthcare Partners,Matrix Partners China,Northern Light Venture Capital,Wu Capital,CDG Capital,Cenova Capital,among others. BBM has offices,Research Centers,Development & Manufacturing center and GMP production facility in North Carolina,Shanghai,Hong Kong and Beijing.

BBM is founded by the famous scientist Dr Xiao Xiao,who currently serves as the President and CSO of the company. Dr Xiao is an expert in the gene therapy field with over 35-year experience in engineering AAV. Dr Xiao has contributed to the development of novel gene therapy strategies for inherited muscular dystrophy,hemophilia and lysosomal metabolism diseases. He also works on developing of novel AAV vectors and scaling-up production of rAAV. His groundbreaking work has been developed into the first gene therapy clinical trial for DMD in the United States. This work in Bamboo Therapeutics was acquired by Pfizer for over US$ 650 million. Prior to that,Dr Xiao co-founded AskBio,which was acquired by Bayer for over US$ 4 billion in October 2020.

With deep knowledge of disease mechanism and AAV science,BBM challenges the inevitability of genetic diseases and aims to bringing patients significant and long-lasting improvements to their life quality.

BBM has established a robust pipeline to address a spectrum of life-threatening diseases,including genetic diseases,neuromuscular & neurological diseases,autoimmune,malignant tumors,etc. Its core competencies include rAAV viral vector development and gene therapy manufacturing. The experienced team has built the entire workflow of AAV drug development consisting of viral vector design and optimization,bioprocessing development and scalable manufacturing up to 500L.

The IND approval of BBM-H901 is a significant milestone for the company. It will open a "new chapter" for the story of gene therapy in China.

BBM has the vision of "To be the leader of China and global Gene Therapy". BBM is committed to making high-quality Gene Therapy available and affordable for patients around the world.

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