BBM-D101

今日（4月18日），中国国家药监局药品审评中心（CDE）官网刚刚公示，信念医药在研的1类新药BBM-D101注射液获批临床，拟开发治疗杜氏肌营养不良（DMD）。公开资料显示，这是一款腺相关病毒（AAV）基因治疗药物。本次是该产品首次在中国获批IND。截图来源：CDE官网DMD是一种由于抗肌萎缩蛋白（dystrophin）基因突变导致的X连锁隐性遗传肌肉疾病。患者症状通常出现于6岁之前甚至婴幼儿时期，多数患者在10-12岁逐渐丧失行走能力，30岁左右因心脏和/或呼吸功能衰竭失去生命，临床亟需创新治疗方案。据信念医药公开资料介绍，BBM-D101注射液是一款在研的AAV基因治疗药物。通过单次静脉输注，基于工程化AAV载体，将优化的基因表达盒递送到全身肌肉，以期实现“一次给药、长期有效”地治疗DMD。该产品使用了工程化衣壳，其肌肉组织递送效率更高，临床拟用剂量较低，有望具有良好的安全性。2024年7月，BBM-D101注射液的研究者发起的临床研究（IIT）启动；2024年11月，该产品获得美国FDA授予孤儿药资格（ODD）和儿科罕见病资格（RPDD）；2025年1月，该产品在美国获批IND。本次BBM-D101注射液在中国获批IND，意味着该产品的临床研究进程迎来新的进展。本次获批的临床试验是一项开放、单次给药临床研究，旨在评估静脉输注BBM-D101注射液在4~9岁DMD男孩中的安全性和有效性。公开资料显示，信念医药致力于为严重遗传疾病和慢性疾病提供更加有效的创新性基因疗法。该公司已经建立了丰富的研发管线，治疗领域覆盖血友病、杜氏肌营养不良、帕金森病、骨关节炎等，多个产品管线已经进入临床研究阶段或IND申报阶段，其针对血友病B的基因疗法新药已于近日获中国国家药品监督管理局（NMPA）批准上市。参考资料：[1]中国国家药监局药品审评中心（CDE）官网. Retrieved Apr 18，2025, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c[2]信念医药杜氏肌营养不良症基因治疗药物BBM-D101注射液IND获NMPA受理. Retrieved Feb 5，2025, From https://mp.weixin.qq.com/s/IOPa-BMDszmt0tMRYJCdSA[3]中国首个血友病B基因治疗药物信玖凝®（波哌达可基注射液）正式获批. Retrieved Apr 10，2025, From https://mp.weixin.qq.com/s/mhH10NpexfLPeFP27O5JSQ免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

Shanghai, China, April 18th, 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-D101, the company’s Duchenne Muscular Dystrophy (DMD) gene therapy candidate. Thus far, the IND for BBM-D101 has been approved by both NMPA and the Food and Drug Administration (FDA) of America. Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, “We are pleased to share the good news of the NMPA’s IND clearance of BBM-D101. We appreciate the NMPA’s review of the IND and look forward to continuing to work in collaboration with the agency to carry out clinical trial. Previously, it has been obtained IND clearance by FDA. We will accelerate the progress of clinical research to bring new treatment options to DMD patients as soon as possible.” This clinical trial is an open label and single-dose clinical study, which was designed to evaluate the safety and efficacy of a single intravenous infusion in DMD patients aged 4-9 years old. Due to engineered capsid, BBM-D101 has a higher efficiency to transduce muscle tissue. Therefore, the proposed clinical dosage is lower than the approved similar adeno-associated virus (AAV) gene therapy products, and BBM-D101 is expected to have good safety. 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 worldwide [1], and nearly 30% of them have new gene mutations [2]. The estimated number of DMD patients in the United States is less than 50,000 [3]. 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 old [4]. 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, thereby achieving "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 Qualification (ODD) and Pediatric Rare Disease Qualification (RPDD) by the FDA. January 2025, BBM-D101 received FDA clearance of an Investigational New Drug (IND) application. April 2025, BBM-D101 received NMPA clearance of an Investigational New Drug (IND) application. [1] https://www-ncbi-nlm-nih-gov.libproxy1.nus.edu.sg/pmc/articles/PMC10330733/ [2] ttps://www.ncbi.nlm.nih.gov/books/NBK482346/ [3] https://rarediseases-info-nih-gov.libproxy1.nus.edu.sg/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 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-D101 described herein has not been approved for marketing.