Gene therapy digest - 2021

Gene therapy digest - 2021

NMPA granted first IND approval for Belief Biomed’s gene therapy

BBM-H901 (AAV gene therapy; IND approved) – Belief Biomed

  • IND approval is for BBM-H901, an IV infusion gene therapy for Hemophilia B in China. Phase 1/2 trial will be initiated shortly
  • BBM-H901 is a bio-engineered adeno-associated viral (AAV) vector utilizing, novel recombinant AAV capsid containing a codon-optimized human factor IX gene under the control of a liver-specific promoter
  • Original IND application to NMPA in Apr 2021 and received clearance on Aug 6, 2021
  • Investigator Initiated Trial (IIT) of BBM-H901 is one of the earliest clinical trials for AAV Gene Therapy carried out in China
  • 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 (For full story click here)

Belief Biomed's gene therapy for Hemophilia B received NMPA IND approval: First intravenous infusion gene therapy in China

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CI Scientists Commentary:

  • Recombinant factor IX replacement therapy remains the standard treatment for Hemophilia B
  • Disadvantages of replacement therapies are
    • Development of inhibitors (neutralizing antibodies) leading to insufficient treatment effects
    • Low half-life
  • Gene therapies are being investigated which allow for long-term expression of the missing or lacking factor IX in the blood without frequent medical intervention
  • With hemophilia pipeline getting crowded with multiple gene therapies both in late and early-stage, there are uncertainties on how long will the treatment effect last and will it be a one-time treatment

– Dr. Kowndinya, CI Scientists

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