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渤健|速递｜开发创新眼科基因疗法，ViGeneron与渤健达成全球性合作协议

▎本文转自ViGeneron公司新闻稿
2021年1月5日，基因治疗公司ViGeneron宣布与位于美国马萨诸塞州剑桥的渤健（Biogen）公司达成全球性的合作和授权协议，用于开发和商业化一款基于腺相关病毒（AAV）载体的治疗遗传性眼病的基因治疗产品 。双方将利用ViGeneron专有的vgAAV 平台、创新的改造AAV衣壳，通过向眼睛玻璃体内注射，实现视网膜细胞的高效转导 。
“基因治疗已在临床成为现实 。在ViGeneron，我们致力于开发创新的基因疗法，以治疗医疗需求未得到满足的疾病 。此次合作体现了我们的战略，我们既在公司内部针对筛选出的视网膜靶点进行基因治疗产品开发，同时也通过对其他眼科靶点和其他适应症的更多合作计划，最大限度地利用我们的专有技术平台 。” ViGeneron公司联合创始人兼首席执行官徐曼博士这样说道，“ViGeneron在视网膜基因治疗方面公认的专业能力以及渤健领先的研究、药物开发和商业化经验是一个强有力的组合，我们相信这将使我们能够为有需要的患者提供更多创新的基因疗法 。”
渤健|速递｜开发创新眼科基因疗法，ViGeneron与渤健达成全球性合作协议
文章图片

▲ViGeneron公司联合创始人兼首席执行官徐曼博士（图片来源：ViGeneron公司官网）
在此次合作中，ViGeneron将优化和验证用于治疗遗传性眼病的体外治疗候选药物（靶点未公开） 。渤健有权在协议生效日期后的两年内增加一个额外的靶点 。两家公司将在体内概念验证（POC）阶段合作 ，渤健将负责这些候选药物后续所有的开发和商业化 。
ViGeneron将从渤健获得预付款和双方商定的工作计划的研发经费 。此外，ViGeneron将会获得开发、监管审批和商业化过程中的里程碑付款，并且也会获得合作产品所带来的净销售额的一定比例的分级提成 。
关于ViGeneron
ViGeneron signs global collaboration agreement for ophthalmic gene therapy development
January 5th, 2021 –– ViGeneron GmbH, a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. (Nasdaq: BIIB, Cambridge, Mass., USA) to develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors to treat inherited eye diseases. The companies will use ViGeneron’s proprietary vgAAV, novel engineered AAV capsids, to efficiently transduce retinal cells via intravitreal injections.
“Gene therapy has become a clinical reality. At ViGeneron, we are dedicated to developing innovative gene therapies to treat diseases with high unmet medical need. This collaboration exemplifies our strategy to develop in-house programs for selected retinal targets, while maximizing our proprietary technology platforms with additional collaboration programs for other targets in ophthalmology and further indications.” said Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron. “ViGeneron’s recognized expertise in retinal gene therapy together with Biogen’s leading research, drug development and commercialization experience is a powerful combination that we believe will allow us to deliver more novel gene therapies to patients in need.”
Within the collaboration, ViGeneron will optimize and validate in vitro therapeutic candidates for an undisclosed target to treat inherited eye disease. Biogen has the right to add an additional reserved target within two years after the effective date. The companies will work together on the in vivo proof of concept (POC). Biogen will be responsible for all further development and commercialization of the selected therapeutic candidates.
【渤健|速递｜开发创新眼科基因疗法，ViGeneron与渤健达成全球性合作协议】ViGeneron will receive from Biogen an upfront payment and R&D funding for the mutually agreed workplan. In addition, ViGeneron will be eligible to receive development, regulatory and commercial milestone payments, and will also be eligible to receive tiered royalties on net commercial sales of products arising from the collaboration.

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