Shanghai, China, March 27th, 2025 —— Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that it has received National Medical Products Administration (NMPA) clearance of an Investigational New Drug (IND) application for BBM-A101, a gene therapy drug for knee osteoarthritis (KOA).

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, "China's aging population is accelerating, leading to a growing burden of chronic disease healthcare. Building on its extensive experience in the research and production of gene therapies for rare diseases, BBM is actively expanding its development of gene therapies for chronic diseases. Utilizing an AAV vector to deliver an optimized human anti-inflammatory protein expression cassette to patients' knee joints for sustained expression, BBM-A101 Injection holds the potential to achieve 'single-dose, long-term efficacy' in treating the condition. This approval marks a significant milestone for BBM. We will accelerate clinical development to bring this innovative treatment to patients as soon as possible!".

About Knee Osteoarthritis

Knee Osteoarthritis (KOA) is a chronic, progressively degenerative joint disease characterized by inflammation, subchondral bone remodeling, and articular cartilage damage^[1], which significantly restricts patients' mobility. As of 2020, the global number of osteoarthritis patients has risen to 595 million, accounting for approximately 7.6% of the world's population, and this figure is projected to reach 642 million by 2050^[2].

Current clinical treatments for KOA primarily rely on pharmacotherapy and surgical intervention. However, the adverse effects and long-term risks associated with certain medications limit their clinical application. Meanwhile, total knee arthroplasty is typically reserved for end-stage severe KOA. For the large population of patients in the mid-stage of disease progression who do not meet surgical criteria, there remains a critical unmet medical need for a treatment that offers both long-term efficacy and safety.

About BBM-A101

BBM-A101 utilizes a proprietary adeno-associated virus (AAV) gene delivery vector and an optimized human gene expression cassette developed independently by BBM. Administered via intra-articular injection, the therapy enables sustained expression of anti-inflammatory protein within the joint cavity. This mechanism is expected to alleviate intra-articular inflammation, reduce pain, and improve knee joint function in KOA patients, thereby slowing disease progression and ultimately enhancing treatment outcomes.

The investigator-initiated clinical trial (IIT) for BBM-A101 Injection in knee osteoarthritis is progressing steadily. Preliminary IIT data have demonstrated favorable safety and efficacy profiles. The upcoming Phase I study will further evaluate the safety, tolerability, and efficacy of BBM-A101 Injection in patients with moderate KOA (Kellgren-Lawrence Grade 2 or 3).

[1]   Kumar S, Sugihara F, Suzuki K, Inoue N, Venkateswarathirukumara S. A double-blind, placebo-controlled, randomised, clinical study on the effectiveness of collagen peptide on osteoarthritis. J Sci Food Agric. 2015 Mar 15;95(4):702-7.

[2]   GBD 2021 Osteoarthritis Collaborators. Global, regional, and national burden of osteoarthritis, 1990-2020 and projections to 2050: a systematic analysis for the Global Burden of Disease Study 2021. Lancet Rheumatol. 2023 Aug 21;5(9):e508-e522.

About Belief BioMed

Belief BioMed Inc. (BBM) is a global biotech company that integrates the research and development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for severe genetic and chronic diseases through safe and efficient viral vector technology. BBM has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established a commercial production platform for gene therapy drugs. The company has been building up its capabilities in a variety of fields including novel AAV capsids targeting different tissues, efficient transgene expression cassette design, and advanced clinically applicable vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of unmet clinical needs in different therapeutic areas such as hemophilia, DMD, Parkinson's disease, osteoarthritis, etc. Several product pipelines have entered clinical studies or submitted IND filings. The NDA of a gene therapy for the treatment of adult patients with hemophilia B, has been accepted by the NMPA of China. For more information, visit www.beliefbiomed.com.

Statement

This information is only for the purpose of introducing the company's event and information on that date, and is not intended to promote any company's products and/or services, nor should it be construed as providing any advice or recommendation on the selection of any drugs, medical devices and treatment options.

For information about any company products, diseases and/or treatments, please consult a healthcare professional.

BBM-A101 described herein has not been approved for marketing.