Shanghai, China, August 14, 2026 — Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that it has received Fast Track Designation (FTD) from U.S. Food and Drug Administration (FDA) for BBM-D101, the company’s Duchenne Muscular Dystrophy (DMD) gene therapy candidate. This marks another recognition by regulatory authorities of BBM-D101's clinical value.

FTD is an FDA designation for investigational drugs designed to speed up the development and review of therapies for serious or life-threatening diseases, especially those that show promise in addressing unmet medical needs. To receive FTD, a therapy must target a serious condition and show, through preclinical and/or clinical data, the potential to fill that unmet need. Recipients of FTD gain access to a range of FDA support mechanisms during their development and regulatory review.

Previously, BBM-D101 received both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. FDA in November 2024, followed by FDA clearance of its Investigational New Drug (IND) application in January 2025. Combined with this latest FTD, these milestones mark the sustained, high-level recognition by international regulatory authorities of Belief BioMed's R&D capabilities and clinical value in the field of gene therapy. These designations have the potential to provide strong support for BBM-D101's subsequent clinical development, regulatory review, and commercialization, and may ultimately accelerate patient access to this therapy.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle disease caused by mutations in the dystrophin gene. There is about 1 DMD patient per 5,000 male infants worldwide¹, and nearly 30% of them have new gene mutations². The estimated number of DMD patients in the United States is less than 50,000³. The symptoms of DMD usually appear before the age of 6 years old or even in infancy. Most patients gradually lose the ability to walk at the age of 10-12 years old and lose their lives due to cardiac and / or respiratory failure at the age of 30 years old⁴. Innovative treatment options are urgently needed.

About BBM-D101

BBM-D101 is an AAV-based gene therapy with independent intellectual property rights owned by BBM. Based on engineered AAV vector, it is single intravenously administered to deliver the optimized gene into muscles of whole body, and is therefore expected to achieve "one dose, long-term effect" treatment of DMD. The production of BBM-D101 uses the 500L serum-free suspension culture process independently developed by the company, which meets the requirements of the Good Manufacturing Practice of Medical Products (GMP).

Since July 2024, the Investigator Initiated Trial (IIT, NCT06641895) of BBM-D101 has been on-going. November 2024, BBM-D101 was granted Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (RPDD) by the U.S. FDA. January 2025, the IND application for BBM-D101 was cleared by the U.S. FDA. April 2025, the IND application was accepted by the National Medical Products Administration (NMPA) of China. August 2025, the first participant was dosed in the registrational clinical study of BBM-D101 for the treatment of DMD. August 2026, BBM-D101 received Fast Track Designation (FTD) from the U.S. FDA.

References

[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10330733/

[2] https://www.ncbi.nlm.nih.gov/books/NBK482346/

[3] https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy

[4] https://www.genome.gov/Genetic-Disorders/Duchenne-Muscular-Dystrophy

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 Biologics License Application (BLA) of a gene therapy for the treatment of adult patients with hemophilia B, has been approved by the NMPA of China.

Statement

1.   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.

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

3.   BBM-D101 described herein has not been approved for marketing.