Ohr Pharmaceutical, Inc.

The partnership aims to leverage both companies’ expertise in gene therapy technology. Credit: illustrissima/Shutterstock. Asklepios BioPharmaceutical (AskBio), a subsidiary of Bayer , has entered a strategic partnership with China-based Belief BioMed (BBM) to advance the development of new gene therapies. The partnership aims to leverage both companies’ expertise in gene therapy technology, particularly for conditions that can be treated through a liver-targeted approach. Using viral vector technology, BBM unifies the development, production and clinical usage of experimental gene therapy products for severe hereditary and chronic illnesses. The business has created advanced vector technology and set up a platform for the commercial manufacture of gene therapy medications in China. AskBio chief scientific officer Mansuo Shannon stated: “AskBio takes a collaborative approach when developing new gene therapies, and we look worldwide to use the most promising science to realise our goals. See Also: Biologic API Manufacturing Companies (Protein and Peptide) in Contract Manufacturing for the Pharmaceutical Industry Bavarian Nordic gains US contract for additional mpox vaccine “The potential of BBM’s next-generation capsid technology, together with the work we are conducting at AskBio, is promising. We are looking forward to working closely with the team at BBM.” Belief BioMed’s new drug application for a haemophilia B treatment was accepted by the China National Medical Products Administration in July 2024. This application is the first submitted in China for a gene therapy product for an inherited genetic disease. BBM has developed new engineered adeno-associated virus vectors. These products have demonstrated the potential to reduce immunogenicity and increase transduction efficiency in human and non-human trials. Bayer Pharmaceuticals business development and licensing head Juergen Eckhardt stated: “Gene therapy has some of the greatest potential in modern medicine, particularly from a technical perspective. “One path to success lies in collaborations such as this and in bringing together experts with broad expertise and experience. We are excited for AskBio’s new collaboration with BBM and the advancements it may one day bring to patients.” In January 2023, AskBio entered a research partnership and option agreement with ReCode Therapeutics to assess its single-vector gene-editing platform . 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

Belief BioMed and AskBio will work together to advance potential gene therapies in diseases with high unmet medical need, using a liver-targeted approach. SHANGHAI, Sept. 25, 2024 /PRNewswire/ -- Belief BioMed Inc. (BBM), a biotech company focusing on innovative gene therapies, today announced a new strategic collaboration with Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, to explore the potential for new gene therapies. Under the terms of the contract, the companies will combine efforts and experience in gene therapy technology to explore potential therapies in diseases that may be treatable using a liver-targeted approach. Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, "Focusing on gene therapy, Belief BioMed has achieved exciting results in research, development and production since its inception. The company's proprietary recombinant adeno-associated virus (rAAV) vector system has also been widely recognized by the industry. Our strategic partnership with AskBio is based on our shared vision, mutual trust and complementary resources and strength. With our joint effort we will be able to bring more innovative treatments in a timely manner to patients around the globe." With global headquarters in North Carolina, US, AskBio is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications. The company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. Gustavo Pesquin, Chief Executive Officer, AskBio, said, "This strategic collaboration with BBM is an excellent example of how we work at AskBio and is particularly special given the extraordinary contributions Dr. Xiao Xiao has made to the field while at BBM and before as a co-founder of AskBio. Collaborating with innovative, like-minded partners with complementary gene therapy expertise enables us to find the best way forward for our pipeline assets and bolster our efforts to advance new therapies for conditions with significant unmet need." Dr. Jane Zheng, Co-founder and Chief Executive Officer of Belief BioMed, said, "The high-quality, steady development of Belief BioMed in recent years is not only attributable to the dedication and unremitting efforts of our employees, but also to the strong support given by our external partners. Belief BioMed has always been actively building open, collaborative, and win-win partnerships, just like the cooperation between the two companies this time. Under the framework of this novel and progressive collaboration, we look forward to working together with AskBio to address a wider range of unmet medical needs, and at the same time, taking another solid step towards the goal of Belief BioMed: rooted in China, serving the world." Belief BioMed is a biotech company that integrates the research & development, manufacturing, and clinical application of gene therapy products. Its pipeline covers a wide range of therapeutic areas that include hemophilia, Duchenne Muscular Dystrophy (DMD), Parkinson's disease, and osteoarthritis. This collaboration will further strengthen the company's core competitiveness in the field of gene therapy, and create a solid foundation for bringing additional new innovative gene therapies to patients worldwide. About Belief BioMed Belief BioMed Inc. (BBM) is a 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 in China. 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 . Forward-looking statements The forward-looking statements made in this article only relate to events or information as of the date when the statements are made in this article. Except as required by law, we undertake no obligation to update or publicly revise any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. All statements in this article are made on the date of publication of this article and may change due to future developments. SOURCE Belief BioMed WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

关注并星标CPHI制药在线 2024年6月24日，信念医药集团（简称BBM或“信念医药”）发布重要公告：公司核心候选产品BBM-H901注射液，在一项由研究者主导的临床试验（IIT，登记号NCT04135300）中，针对B型血友病成年患者出血控制的长期追踪研究结果，已在全球瞩目的第32届国际血栓与止血学会年会（ISTH）上以口头报告的形式向业界揭晓。紧接着，信念医药于同年7月24日官方宣布，中国国家药品监督管理局（NMPA）已正式接纳了BBM-H901注射液作为治疗血友病B成年患者的新药上市申请（NDA），标志着其正式步入了监管审批流程。 BBM-H901注射液，作为信念医药独立研发并制造的首款旗舰级产品，同时也是中国首个针对遗传性疾病申请新药上市的基因治疗药物，其重要性不言而喻。这一历史性的突破，预示着BBM-H901或将开创先河，成为中国首个获批用于B型血友病治疗的重组腺相关病毒（AAV）基因疗法，为血友病患者带来全新的治疗希望，并预示着中国医药行业正逐步迈入基因疗法的新纪元。 BBM-H901注射液，蕴含了信念医药团队的深厚智慧与不懈努力，它是一款采用优化后的人凝血因子Ⅸ（FIX）基因表达系统，封装于重组腺相关病毒载体中的创新基因治疗药物。这一产品的研发与申请上市，不仅彰显了信念医药在基因治疗领域的地位，也为中国乃至全球的生物医药产业树立了新的里程碑。 一、血友病——碰不得的“玻璃人” 血友病，作为一种具有显著遗传特性的出血障碍疾病，其遗传模式遵循X染色体连锁隐性遗传规律。依据遗传学上基因突变的差异性，血友病被细分为两大类型：血友病A，即凝血因子VIII（也称为抗血友病球蛋白A）缺乏症；以及血友病B，即以凝血因子IX缺乏为主要特征的疾病。在患者群体中，血友病A占据主导地位，占比约为80%至85%，而血友病B则占据剩余约15%至20%的份额。尤其值得注意的是，在男性群体中，两种疾病的发病率分别为血友病A约1/5000，血友病B则显著降低至约1/25000。 血友病的核心症状在于患者难以控制的出血倾向，即便是轻微的碰撞或创伤也可能引发严重的出血事件。这一疾病主要侵扰男性群体，其临床表现多样，从自发性出血到轻度外伤、小型手术（诸如拔牙、扁桃体摘除术）后的持续出血均有涉及。病情的严重程度与凝血因子的缺乏程度紧密相关，轻度患者可能在经历较大创伤或手术后才显露出出血问题，而重度患者则可能遭遇无诱因的自发性出血，此类出血常见于关节、肌肉深层及内脏器官如胃肠、泌尿道甚至脑部，不仅带来剧烈疼痛，还可能引发长期残疾乃至威胁生命。 追溯血友病的根源，其源于基因层面的异常变异。具体而言，血友病A患者是因X染色体上的F8基因发生突变所致，而血友病B则与F9基因的变异紧密相关。这些关键基因负责编码凝血过程中不可或缺的凝血因子，任何形式的突变都可能导致凝血因子结构或数量的异常，进而扰乱血液的凝固机制。在遗传模式上，血友病严格遵循X染色体连锁隐性遗传，意味着男性因仅拥有一条X染色体而更易受到影响，而女性则需从双亲处各继承一条致病变异的X染色体方能显现疾病状态。 二 、关于BBM-H901 BBM-H901注射液，作为信念医药精心研发的创新产品，其问世标志着我国在B型血友病治疗领域的重大突破。这款注射液的独特之处在于，它能通过静脉给药途径，将编码人凝血因子IX的基因有效送入B型血友病患者的体细胞中，实现基因的持续表达，显著提升并稳定保持患者的凝血因子IX水平，从而为成年患者构建起出血事件的预防性屏障。 尤为值得一提的是，BBM-H901注射液的临床研究成果在2022年取得了国际认可，其研究报告相继在《柳叶刀-血液病学》及《新英格兰医学杂志》这两大世界顶级医学期刊上刊发，不仅彰显了研究的科学价值，也体现了国际医学界对该产品治疗潜力的广泛认可。同年，这一创新疗法更是被国家药品监督管理局药品审评中心高度评价，纳入“突破性治疗品种”名单，预示着其有望加速通过审批流程，尽早惠及更多患者。 此外，在享有盛誉的第32届国际血栓与止血学会大会（ISTH）上，BBM-H901注射液的临床试验数据作为口头报告正式发布，进一步向全球展示了其在B型血友病治疗上的显著疗效与安全性，为全球血友病治疗领域注入了新的活力与希望。 背景： 本研究由中国医学科学院血液病医院发起，是一项单中心、单臂、探索性研究，旨在评估单次静脉输注BBM-H901注射液在中国B型血友病患者（FIX≤2%，年龄≥18岁）中的安全性和有效性。2019年10月至2021年1月，10例受试者成功入组并且完成BBM-H901注射液（5E12 vg/kg）的单次输注治疗。其治疗后截止最后1例受试者访视1年的数据已于2022年5月发表在《柳叶刀-血液病学》上。此次ISTH大会主要公布这10例受试者在BBM-H901注射液输注后，长期随访3.0-4.5年的安全性及疗效数据。 结果： 10例受试者随访3.0-4.5年，中位时间为176周（153-235周）。所有受试者最后一次随访FIX活性的平均值（±SD）为43.17 (±26.91) %（一期法Actin-FSL）。 在接受BBM-H901注射液治疗后，各时间点平均FIX活性＞30%（一期法Actin-FSL）。 长期随访3.0-4.5年期间，9例受试者FIX持续稳定较高表达，未发生出血事件，年化出血率（ABR）为0，且完全停止了外源性FIX产品的替代治疗。 研究期间，无死亡、无严重不良事件、无血栓栓塞事件、无FⅨ抑制物阳性的不良事件发生，长期随访期间无转氨酶异常的不良事件发生。 结论： 本研究显示，BBM-H901注射液安全性良好，载体衍生的FIX活性水平长期持续稳定表达，并可显著降低出血事件，极大减少B型血友病患者对外源性FIX产品（凝血酶原复合物、凝血因子IX）替代治疗的需求。 三、基因疗法现状与未来 BioMarin的Roctavian，作为全球血友病A治疗领域的创新基因疗法，于2022年8月率先在欧盟市场取得了商业化许可，随后又在次年6月29日赢得了FDA的青睐，在美国成功上市。尽管外界，包括某些国际金融机构，曾预测其年度销售巅峰可达约22亿美元，且BioMarin内部对其2023年的销售目标设定为0.5亿至1.5亿美元，但现实数据却显示，Roctavian在当年的销售额仅为350万美元，这一成绩远低于先前的所有预计。 另一方面，针对血友病B的全球首款基因疗法Hemgenix，已于2022年在美国通过FDA审核上市，其令人瞩目的标价——350万美元，一度将全球最昂贵药物的纪录拔高。不过，有观点提出，若该疗法能有效缓解病情，相较于某些血友病患者一生可能累积的1500万至2000万美元治疗及护理费用而言，其性价比依然显得颇为可观。2023年6月，随着CSL Behring与uniQure联合宣布，首位B型血友病患者接受了Hemgenix的治疗，这一疗法也正式开启了其商业化应用的新篇章。 当前，无论是Roctavian还是Hemgenix，均处于其商业化征程的初期阶段，正积极寻求更广泛的市场覆盖。 值得注意的是，2024年7月25日，辉瑞公司携其针对血友病B的基因疗法再传喜讯，继在加拿大与美国获得批准后，该疗法又在欧洲市场赢得了上市许可。同时，辉瑞还宣布了其在研的另一款针对血友病A的基因疗法——giroctocogene fitelparvovec的3期临床试验（即AFFINE研究）取得了重大成功。针对血友病A，AFFINE研究显示在预防中度至重度血友病A成人患者出血方面展现出了比传统替代预防疗法更为显著的疗效。此疗法由辉瑞与Sangamo Therapeutics共同研发，AFFINE试验的积极结果公布后，Sangamo Therapeutics股价在早盘即实现了高达124%的飙升，彰显了市场对这一突破性疗法的高度认可与期待。 Giroctocogene fitelparvovec，作为一款前沿的基因治疗候选药物，集成了创新的AAV6病毒封装技术和经优化以去除B结构域的人类凝血因子FVIII基因。此疗法的核心愿景是通过单次治疗，赋予患者长期稳定生成FVIII的能力，进而有效预防出血事件，并大幅削减对传统静脉输注预防性治疗的依赖。该疗法已赢得美国FDA的快速通道认证、RMAT（再生医学先进疗法）标签以及孤儿药地位，同时在欧盟也获得了孤儿药认证。 2024年7月26日，基因治疗领域再传佳音。欧洲药品管理局（EMA）正式授予Durveqtix（fidanacogene elaparvovec）有条件上市许可，该产品由辉瑞研制，是基于AAV载体的革命性基因疗法，专为中度至重度血友病B且不受AAV Rh74血清型抗体影响的患者量身打造。值得一提的是，该疗法已在今年早些时候分别在加拿大和美国获得上市批准，其中美国FDA批准的商品名为Beqvez。 在国内市场，BBM-H901正蓄势待发，有望成为我国首发的基因治疗药品，这一里程碑式的成就不仅标志着我国在基因治疗领域的重大突破，也为患者提供了更加多元化的治疗选择。 END 【智药研习社直播预告】 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~