Spark Therapeutics, Inc.

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。近日，一家西班牙CGT公司 SpliceBio 宣布完成 B 轮 1.35 亿美元融资。此次融资由 EQT Life Sciences 和 Sanofi Ventures 联合领投，Roche Venture Fund、New Enterprise Associates 等 7 家机构跟投。融资将用于推进眼病基因疗法 SB - 007 的 1/2 期临床研究，加速眼科、神经学等领域 AAV 基因治疗管线开发，助力 SpliceBio 突破技术与临床转化瓶颈 。SpliceBio 是一家来自西班牙巴塞罗那的临床阶段基因药物公司，聚焦大基因突变导致的疾病，布局眼科、神经病学及其他未公开治疗领域，开发下一代 AAV 基因疗法 。当前，众多基因治疗开发者依赖腺相关病毒（AAVs）递送遗传物质，但 AAVs 因载体容量小，无法携带大型矫正基因，使诸多疾病难以通过常规基因疗法治疗。以杜氏肌营养不良为例，编码肌肉保护蛋白 dystrophin 的基因过大，部分公司只能开发产生截短蛋白的基因疗法，然而截短蛋白功能往往不及完整蛋白，疗效受限。SpliceBio 另辟蹊径，采用 “双 AAV + 蛋白剪接” 策略：用两个独立 AAV 载体将大型基因片段送入细胞，借助工程化剪接分子，在细胞内重新组装成全长功能蛋白，突破 AAV 载体容量限制，为大型致病基因相关疾病治疗开辟新路径 。SpliceBio 的技术源于普林斯顿大学研究，创始人兼 CEO Miquel Vila - Perelló 曾在此钻研蛋白设计与工程。公司 2014 年以 ProteoDesign 之名起步，现除眼科（Stargardt 病等）外，还布局神经学及其他未披露治疗领域，构建多元研发管线 。此前，罗氏旗下 Spark Therapeutics 已在 2023 年与 SpliceBio 达成 1.26 亿美元合作，借助其平台攻克基因过大无法适配传统 AAV 载体的难题，彰显行业对 SpliceBio 技术的认可。SpliceBio的核心项目SB - 007主要针对Stargardt 病（目前无获批疗法），Stargardt 病由 ABCA4 基因突变引发，该基因过大无法装入传统 AAV 载体，导致患者渐进性视力丧失。SpliceBio 的 SB - 007 疗法，利用上述创新技术，助力患者体内产生功能性 ABCA4 蛋白。SB - 007 是首款获美国 FDA 批准进入 Stargardt 病临床开发的双 AAV 基因疗法，也拿到英国药品和保健品监管局（MHRA）临床开发许可 。目前已进入 1/2 期临床试验，2025 年 3 月完成首例患者给药。值得关注的是，SpliceBio 并非孤军奋战，欧洲公司 AAVantgarde 也在早期测试中探索类似策略，赛道竞争与协同创新并存，推动 Stargardt 病基因治疗加速发展。研发管线参考资料：https://splice.bio/splicebio-secures-135-million-series-b-financing-to-advance-lead-program-sb-007-in-stargardt-disease-and-expand-pipeline-of-genetic-medicines/-END-扫码参与报名

近日，SpliceBio公司宣布完成1.35亿美元的B轮融资，用于推进其主要管线SB-007的临床开发。本轮融资由EQT Life Sciences和Sanofi Ventures共同领投，其他参与者包括Roche Venture Fund、New Enterprise Associates、UCB Ventures、Ysios Capital、Gilde Healthcare、Novartis Venture Fund和Asabys Partners。SpliceBio成立于2022年，致力于旨在运用蛋白剪接技术克服AAV载体装载空间有限的障碍，开发出“大基因突变相关疾病”的基因疗法。至今，SpliceBio完成了三轮融资，2020年完成由Ysios Capital和Asabys Partners支持的种子轮融资，2022年完成由UCB Ventures 和Ysios Capital领投的5700万美元A轮融资。SpliceBio的蛋白剪接技术基于一种名为Split Inteins实现。内含肽（Inteins）是一种具有自我剪接功能的多肽链，存在于各种真细菌、古细菌和真核生物以及病毒和噬菌体中。但天然Inteins存在一些局限性，不适用于药物开发，SpliceBio的联合创始人则开发了工程化的Inteins—Split Inteins。Split Inteins具有蛋白质自我剪接机制，可将两个独立的蛋白质片段（N端和C端）进行衔接，然后切除期自身，形成完整的功能蛋白。AAV已经被广泛作为基因治疗的递送载体，但装载空间有限（最大不能超过4.7kb），这导致其无法递送一些较大的基因，限制了其应用。SpliceBio的蛋白剪接技术旨在将原本无法装载的大基因进行拆分，然后分别由携带“Split  Inteins”的两个AAV载体装载，AAV载体进入人体内后，由Split Inteins进行蛋白质剪接，将其重新组装成完整的全长蛋白。根据SpliceBio官网披露，目前公司有四条管线，其中最快的为SB-007，目前正在处于Ⅰ/Ⅱ期临床阶段。SB-007是一种针对Stargardt病的双AAV基因疗法。Stargardt病由ABCA4基因突变引起，会导致进行性视力丧失，直至失明。但ABCA4基因长达6.8kb，单靠一个AAV载体无法装载。SpliceBio将ABCA4基因分成两段，分别装载于“携带内含肽”的两个AAV载体，然后通过Split Inteins发挥蛋白剪接作用，最终组装为功能完整的ABCA4蛋白。今年3月，SpliceBio完成了对SB-007的I/II ASTRA首位患者给药，目前该试验正处于剂量递增阶段。在2023年，SpliceBio还与罗氏旗下Spark Therapeutics达成一笔1.26亿美元的合作，以利用其蛋白剪接平台开发针对未公开遗传性视网膜疾病的基因疗法。参考出处：https://www.biospace.com/business/splicebio-nabs-135-million-series-b-to-advance-protein-splicing-medicineshttps://splice.bio/splicebio-secures-135-million-series-b-financing-to-advance-lead-program-sb-007-in-stargardt-disease-and-expand-pipeline-of-genetic-medicines/

Stargardt disease is a genetic retinal condition, resulting in progressive loss of vision and blindness. Credit: Hryshchyshen Serhii/Shutterstock. SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007, its gene therapy candidate for Stargardt disease. Jointly led by Sanofi Ventures and EQT Life Sciences, the funding round also saw participation from Roche Venture Fund and current investors including Novartis Venture Fund, UCB Ventures, New Enterprise Associates and Ysios Capital. The Series B financing will support the ongoing interventional Phase I/II ASTRA trial and the observational POLARIS study of SB-007. The funds will expedite the development of SpliceBio’s AAV gene therapy programme pipeline across fields including neurology, ophthalmology and other undisclosed indications, leveraging the company’s protein splicing platform. Stargardt disease is a genetic retinal condition that occurs due to mutations in the ATP-binding cassette subfamily A member 4 (ABCA4) gene, resulting in progressive loss of vision and eventual blindness. SB-007 has received clearance from the Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) to enter clinical development. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence It aims to deliver a functional copy of the full-length ABCA4 protein, potentially treating individuals with Stargardt disease, irrespective of their specific ABCA4 mutation. Three new members will join the company’s board of directors:  EQT Life Sciences managing director Daniela Begolo, Sanofi Ventures partner Laia Crespo and Roche Venture Fund head Carole Nuechterlein. SpliceBio co-founder and CEO Miquel Vila-Perelló stated: “This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology and beyond. “The support from such high-quality investors underscores the strength of our programmes and our unique protein splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today.” In 2023, SpliceBio signed a licensing agreement with Spark Therapeutics, allowing the latter to use its protein splicing platform and develop a gene therapy for an undisclosed inherited renal disease. 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 You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy . Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now