LEXINGTON, Mass., Sept. 16, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced that the joint steering committee with its collaborator Neurocrine Biosciences, Inc. has selected a development candidate in a gene therapy program for the potential treatment of an undisclosed neurological disease.

马萨诸塞州列克星敦，2024年9月16日（环球通讯社）--致力于推进神经遗传药物的生物技术公司Voyager Therapeutics，Inc.（纳斯达克代码：VYGR）今天宣布，联合指导委员会及其合作者Neurocrine Biosciences，Inc.已在一项基因治疗计划中选择了一名开发候选人，用于潜在治疗未公开的神经系统疾病。

The candidate leverages an intravenously administered, blood-brain barrier-penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform..

候选人利用静脉注射的血脑屏障渗透剂，这种新型衣壳源自旅行者的TRACER™衣壳发现平台。。

Selection of the development candidate triggered a $3 million milestone payment to Voyager, which the Company expects to receive in the fourth quarter of 2024. Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program..

选择开发候选人引发了对旅行者300万美元里程碑式付款，该公司预计将在2024年第四季度收到。根据该计划的进一步推进，旅行者有资格获得额外的未来开发和商业化里程碑付款。。

“This is the third development candidate nominated under Voyager’s collaborations with Neurocrine, following nominations for Friedreich’s ataxia and for GBA1 Parkinson’s disease and other GBA1-mediated diseases,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “These partnered programs provide multiple opportunities for success and complement our wholly-owned pipeline, including our SOD1 ALS gene therapy program, which is advancing towards an expected IND in mid-2025.”.

Voyager首席执行官Alfred W.Sandrock博士说：“这是继Friedreich共济失调、GBA1帕金森氏病和其他GBA1介导的疾病提名后，Voyager与Neurocrine合作提名的第三位发展候选人。”。“这些合作项目为成功提供了多种机会，并补充了我们的全资渠道，包括我们的SOD1 ALS基因治疗项目，该项目正在向2025年年中的预期IND迈进。”。

This program is being developed under the 2023 strategic collaboration agreement between Voyager and Neurocrine Biosciences for research, development, manufacture, and commercialization of certain AAV gene therapy products for programs targeting Parkinson’s disease and other GBA1-mediated diseases and three other undisclosed programs.

该项目是根据Voyager和Neurocrine Biosciences之间的2023年战略合作协议开发的，用于研究，开发，制造和商业化某些AAV基因治疗产品，用于针对帕金森病和其他GBA1介导的疾病的项目以及其他三个未公开的项目。

Under the terms of the 2023 collaboration agreement, for each of the three undisclosed programs, Voyager is eligible to receive up to $175 million in potential development milestone payments plus substantial potential commercial milestone payments, as well as tiered high single-digit to mid-teens royalties on U.S.

根据2023年合作协议的条款，对于这三个未公开的项目，航行者有资格获得高达1.75亿美元的潜在发展里程碑付款，加上大量潜在的商业里程碑付款，以及美国的分层高个位数至十几岁的版税。

net sales and mid-single-digit to low double-digit royalties on ex-U.S. net sales. Neurocrine Biosciences will fund the development of each program..

净销售额和美国境外净销售额的中位至低位两位数特许权使用费。Neurocrine Biosciences将为每个项目的开发提供资金。。

About the TRACER™ Capsid Discovery Platform

关于TRACER™衣壳发现平台

Voyager’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types.

Voyager的TRACER™（通过RNA的细胞类型特异性表达对AAV进行向性重定向）衣壳发现平台是一种广泛适用的基于RNA的筛选平台，可快速发现新型AAV衣壳以实现基因治疗。旅行者利用示踪剂创建了多个新型衣壳家族，在临床前研究中静脉注射后，利用中枢神经系统（CNS）的广泛脉管系统穿过血脑屏障并转导广泛的CNS区域和细胞类型。

In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager’s wholly-owned and partnered gene therapy programs for neurologic diseases..

在跨物种临床前研究（啮齿动物和多种非人灵长类动物）中，静脉内递送示踪剂产生的衣壳导致在相对较低剂量下在整个中枢神经系统中广泛表达有效载荷，从而能够在Voyager全资和合作的神经系统疾病基因治疗计划中选择多种发育候选者。。

About Voyager Therapeutics

关于Voyager Therapeutics

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system.

Voyager Therapeutics，Inc.（纳斯达克股票代码：VYGR）是一家生物技术公司，致力于利用人类遗传学的力量来改变并最终治愈神经系统疾病。我们的项目包括阿尔茨海默病、肌萎缩侧索硬化症（ALS）、帕金森病和其他多种中枢神经系统疾病的项目。

Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc.

我们的许多程序都来自我们的TRACER™AAV衣壳发现平台，我们已将其用于产生新型衣壳并鉴定相关受体，以在静脉内给药后使遗传药物具有高脑渗透性。我们的一些项目是全资拥有的，一些项目正在与合作伙伴合作，包括Alexion，AstraZeneca Rare Disease；诺华制药公司；Neurocrine Biosciences，Inc。；和Sangamo Therapeutics，Inc。

For more information, visit www.voyagertherapeutics.com..

有关更多信息，请访问www.voyagertherapeutics.com。。

Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc.

Voyager Therapeutics®是Voyager Therapeutics，Inc.的注册商标，TRACER™是Voyager Therapeutics，Inc.的商标。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “expect,” “enable,” “future,” or “potential,” and other similar expressions are intended to identify forward-looking statements..

本新闻稿包含1995年《私人证券诉讼改革法案》和其他联邦证券法中安全港条款的前瞻性声明。使用诸如“期望”、“启用”、“未来”或“潜力”等词语以及其他类似表达旨在识别前瞻性陈述。。

For example, all statements Voyager makes regarding the potential to advance Voyager’s SOD1 ALS gene therapy program towards an IND filing in mid-2025, and Voyager’s ability to receive development and commercial milestone payments, tiered royalties on net sales, and program funding under the 2023 Neurocrine collaboration agreement are forward looking..

例如，Voyager关于将Voyager的SOD1 ALS基因治疗计划推进2025年年中IND申请的潜力，以及Voyager根据2023年Neurocrine合作协议接收开发和商业里程碑付款、净销售额分层版税和计划资金的能力的所有声明都是前瞻性的。。

All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected.

所有前瞻性陈述均基于Voyager管理层的估计和假设，尽管Voyager认为此类前瞻性陈述是合理的，但本质上是不确定的。所有前瞻性陈述都受到风险和不确定性的影响，这些风险和不确定性可能导致实际结果与Voyager预期的结果存在重大差异。

Such risks and uncertainties include, among others, the expectations and decisions of regulatory authorities; the timing, initiation, conduct and outcomes of Voyager’s preclinical and clinical studies; the availability of data from clinical trials; the availability or commercial potential of product candidates under collaborations; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; Voyager’s scientific approach and program development progress, and the restricted supply of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER capsid discovery platform; Voyager’s ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the possibility or the timing of Voyager’s receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager’s existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager .

这些风险和不确定性包括监管机构的期望和决定；Voyager临床前和临床研究的时间，启动，实施和结果；临床试验数据的可用性；合作下候选产品的可用性或商业潜力；旅行者合作伙伴履行与旅行者合作协议项下义务的意愿和能力；继续开发旅行者的技术平台，包括旅行者的示踪剂平台及其抗体筛选技术；旅行者号的科学方法和项目开发进展，以及关键研究组件的供应受限；第三方开发的衣壳识别平台可能与旅行者的追踪衣壳发现平台竞争；Voyager创建和保护与TRACER衣壳发现平台相关的知识产权的能力，该平台识别的衣壳以及Voyager管道项目的开发候选物；Voyager收到项目报销、开发或商业化里程碑、期权行使以及Voyager现有许可或合作协议项下其他付款的可能性或时间；Voyager能够根据Voyager可接受的条款与其他方谈判并完成许可或合作协议。

These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made.

这些声明还受到许多重大风险和不确定性的影响，这些风险和不确定性在旅行者最近提交给证券交易委员会的10-K表格年度报告中有所描述。新闻稿中的所有信息均截至本新闻稿发布之日，任何前瞻性声明仅在发布之日起生效。

Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law..

除法律要求外，旅行者无义务因新信息、未来事件或其他原因而公开更新或修改本信息或任何前瞻性声明。。

Contacts

联系人

Trista Morrison, NACD.DC, tmorrison@vygr.com Investors: Adam Bero, Ph.D., abero@kendallir.com Media: Adam Silverstein, adam@scientpr.com

特里斯塔·莫里森，NACD。DC，tmorrison@vygr.com投资者：Adam Bero博士。abero@kendallir.com媒体：亚当·西尔弗斯坦，adam@scientpr.com

Source: Voyager Therapeutics, Inc.

资料来源：Voyager Therapeutics，Inc。