factor IX

中国上海，2024年10月25日 —— 专注前沿基因治疗领域的全球化创新型企业信念医药集团（Belief BioMed，BBM，下称“信念医药”）今日宣布：欧洲药品管理局（EMA）授予其基因治疗药物BBM-H901注射液先进治疗药物分类认证（Advanced Therapy Medicinal Product, ATMP），用于治疗血友病B。 BBM-H901注射液由信念医药自主研发和生产，是一款携带优化人凝血因子Ⅸ（FIX）基因表达盒的重组腺相关病毒（rAAV）基因治疗药物。欧洲药品管理局作为制定全球药品监管标准的重要力量，授予信念医药该项认证，意味着这一突破性研发成果再次获得监管机构的肯定，公司亦将藉此进一步推进全球化战略以及获得欧洲市场认可。 根据欧盟规定，EMA一般将ATMP授予基于基因、组织或体细胞的疗法，这些疗法有望为人类疾病或损伤带来突破性的治疗方案。通过ATMP的主要权益包括：EMA为认证产品的开发、临床以及生产提供一系列监管指南和激励措施，其中还包括降低科学咨询等相关费用。   作为信念医药的重磅产品之一，BBM-H901注射液已经取得诸多瞩目成绩，其临床潜力与学术价值得以持续印证和彰显：该款药物先后被纳为国家药品监督管理局（NMPA）突破性治疗药物（BTD）品种和新药上市申请（NDA）优先审评品种；获得美国食品药品监督管理局（FDA）的孤儿药认定（ODD）以及儿科罕见病资格认定（RPDD）；研究者发起的临床研究（IIT）结果发表于国际权威期刊《柳叶刀-血液病学》1和《新英格兰医学杂志》2，并在2024国际血栓与止血学会（ISTH）大会进行展示3。 注释 1 Xue F et al. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022;9(7): e504-e513. 2 Xue F et al. Total Knee Arthroplasty after Gene Therapy for Hemophilia B.N EnglJ Med.2022 Oct 27;387(17):1622-1624 3 Xue F et al. More than 3 years follow-up after gene transfer with BBM-H901 in adults with Hemophilia B. 2024 ISTH.OC 30.4 向上滑动阅览 关于血友病B 血友病B是一种遗传性凝血功能障碍疾病，由于患者血液中缺乏凝血因子Ⅸ（FIX）所致，其临床表现为反复的、自发性的关节、肌肉和软组织出血，如长时间反复出血，还可能导致患者关节畸形、肌肉萎缩甚至死亡。整体而言，全球血友病患者仍然面临着治疗选择有限、诊疗不规范等挑战，临床亟需更多创新治疗方案。 向上滑动阅览 关于BBM-H901 BBM-H901注射液由信念医药自主研发和生产，以静脉给药的方式将人凝血因子IX基因导入血友病B患者体内持续表达，提高并长期维持患者的凝血因子水平，用于预防血友病B成年患者出血。2022年，BBM-H901注射液被国家药监局药品审评中心纳入“突破性治疗品种”名单。2024年，该款产品成为国内首个递交新药上市申请（NDA）的针对遗传病的基因治疗药物。其研究者发起的临床研究（IIT）的结果先后发表于国际权威期刊《柳叶刀-血液病学》和《新英格兰医学杂志》，并在2024国际血栓与止血学会（ISTH）大会上以口头报告形式展示。另外，BBM-H901注射液治疗儿童血友病B的IIT在2023年正式启动，并于2024年中完成所有受试者给药。 向上滑动阅览 关于信念医药 信念医药集团公司（Belief BioMed Inc.），是一家全球化的集基因治疗产品研发、生产和临床应用为一体的高科技企业。公司致力于通过安全高效的病毒载体技术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。公司研发了数百种载体关键技术，包括HEK293细胞悬浮无血清培养工艺和全层析规模化纯化工艺，并建立了基因治疗药物商业化生产平台。公司在靶向不同组织的AAV新型衣壳、高效的转基因表达盒设计、先进的临床级载体制造工艺等领域进行了全面布局。同时还建立了丰富的研发管线，治疗领域覆盖血友病、杜氏肌营养不良症、帕金森病、骨关节炎等未被满足临床需求的疾病，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的基因疗法的新药上市申请已获中国国家药品监督管理局受理。 更多信息，请访问：www.beliefbiomed.com。 向上滑动阅览 声明 - 本文仅与陈述当日事件及资料有关，不以宣传任何公司产品和/或服务为目的，更不应被理解为就任何药物及治疗方案的选择提供任何意见或建议。 - 如欲了解任何公司产品、疾病和/或诊疗等相关信息，请务必咨询医疗卫生专业人士。 - 本文所述BBM-H901注射液尚未获批上市。 通过点击链接，您可详阅信念医药公众号法律声明

Be Bio’s BE-101 works by inserting the human Factor IX (FIX) gene into primary human B cells. Image credit: Shutterstock/ ardhikauw. Be Biopharma has raised $82m in financing to advance a gene therapy targeting hemophilia B to a Phase I/II trial, whilst also adding an ex-Roche exec who was involved with the launch of a haemophilia A treatment to its roster. The financing had big pharma involvement in the form of Bristol Myers Squibb and Takeda Ventures, with venture capital firms ARCH Venture Partners, Atlas Venture, and RA Capital Management participating. Be Bio said the funds, which were also from Alta Partners and the Longwood Fund, will be allocated to the hemophilia B candidate BE-101 and  a newly unveiled programme for hypophosphatasia called BE-102. Alongside the funds, the US biopharma added Suha Patel and Dr. Kiran Patki to its ranks, who will serve as senior vice president of commercial & franchise strategy and senior vice president of clinical development, respectively. Patel was involved in launching the haemophilia A treatment Hemlibra (emicizumab-kxwh) at Roche’s Genentech, whilst Patki previously worked at rare disease biotech Rally Bio. Be Bio’s CEO Dr. Joanne Smith-Farrell said that “some valued colleagues will be moving on” as the company shifts resources toward product development. Be Bio’s BE-101 is a genetically engineered B cell therapeutic that works by inserting the human Factor IX (FIX) gene into primary human B cells, which allows for continuous expression of active FIX levels with a single infusion. Haemophilia B, which affects around 40,000 globally, is an X-linked recessive bleeding disorder caused by a deficiency in clotting factor IX and has traditionally been treated with regular infusions of the protein. The advancement of gene therapies is changing the treatment paradigm for this disorder, with single infusions reducing the burden on patients taking repeated treatments. There are currently two US Food and Drug Administration (FDA)-approved gene therapies for haemophilia B – CSL Behring’s Hemgenix (etranacogene dezaparvovec) and Pfizer’s Beqvez (fidanacogene elaparvovec ). Be Bio’s candidate has an FDA orphan drug designation and fast track status, which confers benefits such as the possibility of seven years of market exclusivity. See Also: TreeFrog and University of Pittsburgh probe TLS role in immuno-oncology Lilly’s Kisunla does not demonstrate “good value” for UK’s NHS The FDA approved an investigational new drug application (IND) in March this year, greenlighting the company to initiate a trial evaluating the gene therapy. The multi-centre, first-in-human dose escalation study (NCT06611436), called BeCoMe-9, will evaluate the safety and preliminary efficacy of intravenously administered BE-101 in adults with moderately severe to severe haemophilia B. The trial is estimated to enrol 24 patients and has a primary endpoint of adverse events and serious adverse events one year post dose. Smith-Farrell said: “We are eager to clinically demonstrate BE-101’s potential to provide a highly durable FIX replacement therapy for Hemophilia B patients. Additionally, we are excited to advance BE-102 to address the needs of a large patient population with few to no therapeutic options.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Be Bio’s $82 million round announced Tuesday will help the Cambridge, MA-based biotech fund work for two of its drugs in development, including the first-ever B cell therapy to enter human studies for hemophilia B. While the new round is smaller than its $130 million Series B from 2022, the company plans to use the new funding to reach clinical proof-of-concept for its hemophilia B cell therapy and advance a second candidate for hypophosphatasia, a rare genetic disease that disrupts the development of bones and teeth. CEO Joanne Smith-Farrell told Endpoints News the amount was “what we needed to actually get this program all the way to where we’ve got definitive proof-of-concept.” The startup plans to present clinical data next year for hemophilia B. For hypophosphatasia, it expects to seek an IND in 2026, Smith-Farrell said. Companies developing B cell therapies hope they can essentially be long-term protein factories in the body, but without some of the dangers and side effects that come with other cell and gene therapies. The treatments, if they work, could address a range of diseases where people are missing an essential protein. The $82 million round included investors such as ARCH, Atlas Venture, RA Capital and others. Seattle’s Immusoft in October gave a glimpse at results from the first patient to receive a B cell therapy. Immusoft is developing its treatment for mucopolysaccharidosis type I, a genetic disease in which people are missing an enzyme needed to break down sugars. Be Bio has opened enrollment for a Phase 1/2 study of its hemophilia B therapy, BE-101, to adults with moderate-to-severe disease. The experimental treatment is meant to produce Factor IX, a blood clotting factor, without needing preconditioning. The hope is that the therapy, while durable, will also be redosable if needed.