Belief BioMed's BBM-A101 Injection Receives NMPA IND Clearance for Osteoarthritis Gene Therapy

Belief BioMed (BBM), a pioneering biotech company based in Shanghai, China, has achieved a significant milestone with the National Medical Products Administration (NMPA) clearance for its Investigational New Drug (IND) application. This approval concerns BBM-A101, a novel gene therapy aimed at addressing knee osteoarthritis (KOA), a prevalent and progressively debilitating joint condition. The announcement marks a step forward in the development of innovative treatments to meet the needs of patients living with chronic diseases.

Dr. Xiao Xiao, who serves as the Co-founder, Chairman, and Chief Science Officer at Belief BioMed, highlighted the urgency of addressing chronic diseases given China’s aging population. He noted BBM's efforts to expand its expertise from rare disease therapies to chronic conditions like KOA. BBM-A101 utilizes an adeno-associated virus (AAV) vector to deliver a customized human anti-inflammatory protein directly into the knee joint. This technique is designed to ensure prolonged therapeutic effect from a single-dose administration, representing a potential breakthrough in KOA treatment. Dr. Xiao emphasized that this recent approval is crucial for BBM, as it accelerates the journey towards offering this innovative treatment to patients.

Knee osteoarthritis is characterized by joint inflammation, bone remodeling beneath the cartilage, and damage to the cartilage itself, which collectively impede mobility. Globally, the number of individuals affected by osteoarthritis reached 595 million in 2020, accounting for approximately 7.6% of the population. Projections estimate that this figure will increase to 642 million by 2050. Traditional treatments for KOA include medications and surgical procedures, though these are often accompanied by side effects and risks that limit their use. Total knee replacement is generally reserved for the most severe cases. Consequently, there is a substantial unmet need for effective and safe treatments for those in the mid-stages of the disease who are not candidates for surgery.

BBM-A101 represents a cutting-edge approach to KOA therapy. The drug employs a proprietary AAV gene delivery system, which BBM developed independently, to introduce an optimized gene expression cassette into the joint via intra-articular injection. This process allows for continuous production of anti-inflammatory proteins within the joint, aiming to reduce inflammation, alleviate pain, enhance joint function, and slow disease progression.

The clinical development of BBM-A101 is advancing, with an investigator-initiated trial (IIT) already underway. Early results from this trial indicate promising safety and efficacy outcomes. The upcoming Phase I study will expand upon these findings by evaluating the drug’s safety, tolerability, and effectiveness in patients with moderate KOA, classified as Kellgren-Lawrence Grade 2 or 3.

Belief BioMed is committed to advancing the field of gene therapy, focusing on severe genetic and chronic diseases. The company integrates research, development, and clinical application, utilizing viral vector technology to create innovative therapies. BBM has developed numerous proprietary vector technologies and established a commercial production platform for gene therapy drugs. Their research and development pipeline addresses various therapeutic areas, including hemophilia, muscular dystrophy, Parkinson's disease, and osteoarthritis. Several of BBM's products have progressed to clinical trials or submitted IND applications, underscoring their commitment to addressing unmet medical needs.