Apertura基因治疗公司将其可穿透血脑屏障的AAV衣壳授权给多个合作伙伴，推动中枢神经系统治疗的发展-动脉网

Apertura Gene Therapy Licenses Bloodrain Barrier-Penetrant AAV Capsid to Multiple Partners Advancing Central Nervous System Treatments

CISION 等信源发布 2025-08-13 20:00

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Apertura has licensed its novel TfR1 capsid (TfR1 CapX) to partners targeting CNS disorders

Apertura 已将其新型 TfR1 衣壳 (TfR1 CapX) 授权给针对中枢神经系统疾病的合作伙伴。

TfR1 CapX enables broad brain distribution via intravenous dosing, meeting the urgent need for a non-invasive and well-tolerated gene therapy capsid.

TfR1 CapX通过静脉注射实现广泛的大脑分布，满足了对非侵入性且耐受性良好的基因治疗衣壳的迫切需求。

NEW YORK

纽约

，

Aug. 13, 2025

2025年8月13日

/PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions announced several licensing deals with Galibra Neuroscience and Emugen Therapeutics to utilize Apertura's human transferrin receptor 1 capsid (TfR1 CapX) for their central nervous system (CNS) programs.

/PRNewswire/ -- 专注于创新基因治疗解决方案的生物技术公司Apertura Gene Therapy宣布与Galibra Neuroscience和Emugen Therapeutics达成多项许可协议，以利用Apertura的人转铁蛋白受体1衣壳（TfR1 CapX）用于其中枢神经系统（CNS）项目。

A third venture-backed biotechnology company has entered into an Option Agreement with Apertura for multiple CNS indications..

一家第三家风险投资支持的生物技术公司已与Apertura就多个中枢神经系统适应症签订了选择权协议。

'Genetic therapy medicines are often limited by delivery challenges, particularly to crucial areas like the CNS, creating an urgent need for advanced capsids that can effectively access these tissues. Not only is it important to target the CNS but equally important to avoid other tissues to avoid triggering adverse events.

基因治疗药物常因递送挑战而受限，特别是向中枢神经系统等关键区域的递送，这促使了对能够有效到达这些组织的先进衣壳的迫切需求。不仅靶向中枢神经系统很重要，同样重要的是避免接触其他组织，以防止引发不良反应。

We believe Apertura's TfR1 CapX is the best option of a capsid with a promising efficacy and safety profile. Apertura is excited about TfR1 CapX because we recognize the need for more potent and less invasive capsids targeting the CNS.' said Dr. Diego Garzón, Apertura's Vice President of Corporate Development..

我们相信Apertura的TfR1 CapX是具有出色疗效和安全性的一种衣壳的最佳选择。Apertura对TfR1 CapX感到兴奋，因为我们认识到需要更有效且侵入性更小的针对中枢神经系统的衣壳。” Apertura公司企业发展副总裁迭戈·加尔松博士表示。

Apertura's TfR1 CapX builds on the work from the company's academic founder,

Apertura的TfR1 CapX建立在公司学术创始人工作的基础上，

Ben Deverman

本·德弗曼

, Senior Director of Vector Engineering at The Broad Institute of

，布罗德研究所高级主任，负责载体工程的

Harvard

哈佛大学

and MIT. TfR1 CapX is an intravenously (IV) dosed AAV capsid that binds to the human transferrin receptor, TfR1, to cross the blood-brain barrier and broadly transduce tissues in the brain. In extensive preclinical testing conducted independently by several groups, TfR1 CapX has demonstrated a selectivity to the CNS compared to other tissues with more than 50% neuron transduction and 90% astrocyte transduction across different brain regions..

和麻省理工学院。TfR1 CapX 是一种静脉注射 (IV) 的 AAV 衣壳，能够结合人转铁蛋白受体 TfR1，以穿越血脑屏障并广泛转导大脑中的组织。在由多个小组独立进行的广泛临床前测试中，TfR1 CapX 显示出对中枢神经系统的选择性，与其他组织相比，在不同脑区中实现了超过 50% 的神经元转导和 90% 的星形胶质细胞转导。

Galibra Neuroscience will utilize TfR1 CapX for GABA-related disorders, which is the major brain inhibitory neurotransmitter keeping neural communication in check and preventing over-excitation. GABA levels in healthy individuals are highly regulated with over 20 genes responsible for regulating GABA signaling.

Galibra Neuroscience 将利用 TfR1 CapX 来治疗与 GABA 相关的疾病，GABA 是主要的脑抑制性神经递质，负责维持神经通讯的平衡并防止过度兴奋。在健康个体中，GABA 水平受到高度调控，有超过 20 个基因负责调节 GABA 信号传导。

Variants of these genes lead to GABA imbalance, the root cause of several rare pediatric disorders. 'Our work at Boston Children's Hospital demonstrated that the symptoms of a rare GABA disorder, succinic semialdehyde dehydrogenase deficiency (SSADHD) are reversible by gene replacement therapy.' said Dr.

这些基因的变异会导致GABA失衡，这是几种罕见儿科疾病的根本原因。波士顿儿童医院的博士表示：“我们的工作证明了，一种罕见的GABA疾病——琥珀酸半醛脱氢酶缺乏症（SSADHD）的症状可以通过基因替代疗法逆转。”

。

Henry Lee

亨利·李

, MPhil, PhD, Co-Founder of Galibra Neuroscience, Inc. 'Galibra will use Apertura's TfR1 CapX with our proprietary payload for a brain-wide and systemic gene replacement approach. We are excited to use the Apertura technology to safely deliver critical genetic materials to desired cellular targets, resulting in highly effective and safe therapeutic options for patients in need.

，哲学硕士，博士，Galibra神经科学公司联合创始人。 “Galibra将使用Apertura的TfR1 CapX结合我们专有的有效载荷，进行全脑和系统性基因替代方法。我们很高兴使用Apertura技术安全地将关键遗传物质递送到目标细胞，为有需要的患者提供高效且安全的治疗选择。

We look forward to our productive collaboration.' said .

我们期待着富有成效的合作。”他说。

Alexander Rotenberg

亚历山大·罗滕伯格

, MD PhD, Professor of Neurology at Boston Children's Hospital and

，医学博士，波士顿儿童医院神经学教授，

Harvard Medical School

哈佛医学院

and Co-Founder of Galibra Neuroscience, Inc.

Galibra Neuroscience, Inc. 联合创始人

Emugen Therapeutics is developing novel gene therapy solutions to address neurodevelopmental and neurodegenerative diseases. 'We are advancing therapeutic strategies that target the root causes of complex neurological disorders,' said Dr.

Emugen Therapeutics正在开发新型基因治疗方法，以应对神经发育和神经退行性疾病。博士表示：“我们正在推进针对复杂神经系统疾病根源的治疗策略。”

Tyler Brown

泰勒·布朗

, Emugen's Chief Operating Officer. 'TfR1 CapX enables us to overcome one of gene therapy's key challenges—delivering payloads across the blood-brain barrier—by providing a non-invasive capsid platform. We are excited to leverage this technology alongside our transformative gene therapy payloads aimed at restoring neural function and improving outcomes for patients with high unmet medical need.'.

，Emugen的首席运营官。“TfR1 CapX使我们能够通过提供一种非侵入性的衣壳平台，克服基因治疗的关键挑战之一——穿越血脑屏障递送载荷。我们非常兴奋能够将这一技术与我们旨在恢复神经功能并改善高未满足医疗需求患者的治疗效果的变革性基因治疗载荷结合起来。”

Apertura has also entered into an option and licensing agreement with an undisclosed biotechnology company to explore the use of TfR1 CapX with their proprietary payloads targeting CNS disorders. Specific terms of the agreement are not disclosed at this time. 'Across the industry, partners are looking for capsids that not only reach the CNS effectively but also support safe, systemic delivery—without triggering adverse immune responses,' said .

Apertura还与一家未公开的生物技术公司签订了选择权和许可协议，以探索将TfR1 CapX与其专有的针对中枢神经系统疾病的载荷结合使用的可能性。该协议的具体条款目前尚未披露。“在行业内，合作伙伴正在寻找的衣壳不仅要能有效到达中枢神经系统，还要支持安全的系统递送——且不会引发不良免疫反应，”他表示。

Andrew Steinsapir

安德鲁·斯坦萨皮尔

, Director, Gene Therapy Program Lead at Deerfield Management and Acting CTO of Apertura. 'TfR1 CapX was designed with these uses in mind. We're proud to partner with companies large and small who utilize a gene therapy approach, recognize the potential ofTfR1 CapX, and select it as the foundation for their CNS gene therapy strategies.

，Deerfield Management基因治疗项目负责人兼Apertura代理首席技术官。“TfR1 CapX的设计正是基于这些用途。我们很自豪能与大小公司合作，他们采用基因治疗方法，认识到TfR1 CapX的潜力，并将其选为中枢神经系统基因治疗策略的基础。

These collaborations reflect our shared commitment to overcoming delivery challenges and accelerating meaningful therapies for patients.'.

这些合作反映了我们共同致力于克服交付挑战，加速为患者提供有意义的治疗。

About Apertura Gene Therapy

关于Apertura基因治疗

Apertura is a biotechnology company dedicated to advancing gene therapies that address severe, unmet needs. Founded in 2021 on technology from the Broad Institute and with support from Deerfield Management Company. Apertura is based in

Apertura是一家致力于推进基因疗法以满足严重未满足需求的生物技术公司。该公司成立于2021年，基于布罗德研究所的技术，并得到Deerfield管理公司的支持。Apertura总部位于

New York City

纽约市

. For more information, please visit our website at

如需更多信息，请访问我们的网站

www.aperturagtx.com

and follow us on

并关注我们

领英

。

About Galibra Neuroscience

关于Galibra神经科学

Galibra is a gene therapy company aimed to develop cure for neurologic disorders via balancing brain excitation and inhibition. Galibra is co-founded by Drs.

Galibra是一家基因治疗公司，旨在通过平衡大脑的兴奋和抑制来开发治愈神经系统疾病的方法。Galibra由博士们共同创立。

Alexander Rotenberg

亚历山大·罗滕伯格

and

和

Henry Lee

亨利·李

at Boston Children's Hospital and

在波士顿儿童医院和

Harvard Medical School

哈佛医学院

, with a vision to apply cutting edge molecular neuroscience to treat rare genetic neurologic disorders, and then leverage their knowledge and experience to tackle more prevalent neurologic diseases. Please visit their website and social media outlet for details.

，怀着运用前沿分子神经科学治疗罕见遗传性神经系统疾病，并利用他们的知识和经验来应对更为普遍的神经系统疾病的愿景。欲了解详情，请访问他们的网站和社交媒体平台。

https://galibraneuroscience.com/

and

和

LinkedIn.

领英。

About Emugen Therapeutics

关于Emugen Therapeutics

Emugen Therapeutics, LLC is a biotechnology company built on a proprietary, innovative, and broadly applicable suite of payload technologies designed to safely harness the power of next-generation IV-deliverable AAV capsids. Focusing on diseases with well-defined genetic etiologies, cellular mechanisms, or circuit defects, our goal is to develop transformative gene therapies for devastating neurological disorders.

Emugen Therapeutics, LLC 是一家生物技术公司，基于一套专有、创新且广泛适用的有效载荷技术平台，旨在安全利用下一代可静脉注射的AAV衣壳的力量。我们专注于具有明确遗传病因、细胞机制或回路缺陷的疾病，目标是为毁灭性的神经系统疾病开发变革性的基因疗法。

Through our commitment to innovation and collaboration, we strive to redefine what is possible in the ever-changing therapeutics landscape. Launched in 2021 by a renowned group of academic founders and a leadership team from the Broad Institute, .

通过我们对创新和合作的承诺，我们努力重新定义不断变化的治疗领域中什么是可能的。公司于2021年由一群著名的学术创始人和来自布罗德研究所的领导团队创立。

Harvard

哈佛大学

and

和

MIT

麻省理工学院

the company is based in the

公司总部位于

Boston

波士顿

metro area. For more information, please visit