AGC Biologics to Manufacture AAVantgarde’s Dual-Vector Gene Therapies for Inherited Retinal Disorders

AGC生物制品公司将生产AAVantgarde用于遗传性视网膜疾病的双载体基因疗法

November 24, 2025

2025年11月24日

AGC Biologics has entered into a manufacturing agreement with AAVantgarde, a biotechnology company focused on gene therapies for inherited retinal diseases. The collaboration follows AAVantgarde’s recent Series B financing round and supports the development of two investigational therapies using a dual-vector AAV approach..

AGC Biologics与专注于遗传性视网膜疾病基因疗法的生物技术公司AAVantgarde签订了生产协议。此次合作是在AAVantgarde最近完成B轮融资后进行的，并支持使用双载体AAV方法开发两种研究性疗法。

Agreement Covers Two Gene Therapy Candidates in Clinical Development

协议涵盖临床开发中的两种基因治疗候选药物

As part of the agreement, AGC Biologics will provide GMP (Good Manufacturing Practice) manufacturing services for AAVantgarde’s two lead candidates targeting inherited retinal conditions with no currently approved therapies:

作为协议的一部分，AGC Biologics 将为 AAVantgarde 的两种针对目前尚无获批疗法的遗传性视网膜疾病的主打候选药物提供 GMP（良好生产规范）制造服务：

• AAVB-039 for Stargardt Disease:

• AAVB-039用于斯塔加特病：

AAVB-039 is designed to treat Stargardt disease, the most common inherited form of macular degeneration. The condition affects approximately 1 in 6,500 individuals, often causing vision loss in children and young adults. This candidate is being evaluated in a first-in-human Phase 1/2 clinical trial taking place in the United States, United Kingdom, and Europe..

AAVB-039 旨在治疗斯塔加特病，这是最常见的遗传性黄斑变性形式。该病影响约六千五百分之一的人群，常导致儿童和年轻人视力丧失。这款候选药物正在美国、英国和欧洲进行一项首次人体试验的 1/2 期临床试验评估。

• AAVB-081 for Retinitis Pigmentosa Caused by Usher Syndrome Type 1B:

• AAVB-081用于治疗由1B型Usher综合征引起的视网膜色素变性：

AAVB-081 is intended for patients with retinitis pigmentosa resulting from Usher syndrome type 1B, a rare condition that affects around 1 in 50,000 individuals, typically under the age of 10. The therapy is in Phase 1/2 development and is reported to be the first dual AAV gene therapy to enter clinical testing for an ocular disease..

AAVB-081 旨在治疗因 Usher 综合征 1B 型引起的视网膜色素变性的患者，这是一种罕见病，大约每 5 万人中会有 1 人受到影响，通常在 10 岁以下。该疗法处于 1/2 期开发阶段，据报道是首个进入眼部疾病临床试验的双重 AAV 基因疗法。

Dual AAV Vector Strategy Enables Delivery of Large Genes

双AAV载体策略实现大基因的递送

The gene therapy candidates are based on a dual-vector AAV delivery platform that addresses a known limitation in traditional AAV gene therapies, the limited cargo capacity of AAV vectors, which is approximately 4.7 kilobases.

这些基因治疗候选药物基于一种双载体AAV递送平台，该平台解决了传统AAV基因疗法中已知的局限性，即AAV载体有限的载荷容量，约为4.7千碱基。

The approach involves:

该方法包括：

• Dividing the therapeutic gene into two parts.

• 将治疗基因分成两部分。

• Packaging each part into a separate AAV vector.

将每个部分分别包装到单独的AAV载体中。

• Delivering both vectors to the same cell, where the full-length gene is reassembled and can produce the desired protein.

• 将两个载体递送到同一个细胞中，在那里全长基因被重新组装并可以产生所需的蛋白质。

This dual-vector technique is designed to enable the treatment of conditions that cannot be addressed using single-vector systems due to size constraints.

这种双载体技术旨在通过解决单载体系统因尺寸限制而无法处理的条件来实现治疗。

The programs will utilize AGC Biologics’ BravoAAV™ suspension platform, which supports high-yield production processes and aims to streamline development timelines.

这些项目将利用AGC Biologics的BravoAAV™悬浮平台，该平台支持高产量生产流程，并旨在简化开发时间。

Manufacturing to Be Conducted at AGC Biologics’ Milan Facility

生产将在AGC Biologics的米兰工厂进行

Production will take place at the AGC Biologics Milan Cell and Gene Center of Excellence, a site with more than three decades of experience in biologics and a track record that includes 10 product approvals from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).

生产将在AGC生物制品米兰细胞和基因卓越中心进行，该基地拥有三十多年的生物制品经验，并有包括欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）在内的10个产品批准记录。

The BravoAAV platform, developed by AGC Biologics, is intended to support accelerated development, with timelines from gene sequence to clinical material in as few as nine months.

由AGC Biologics开发的BravoAAV平台旨在支持加速开发，从基因序列到临床材料的时间最短可缩至九个月。

“We are delighted to partner with AGC Biologics, a world-class CDMO with deep expertise in viral vector manufacturing. This partnership marks an important milestone for AAVantgarde as we continue to advance our innovative gene therapy pipeline in the clinic,” said Natalia Misciattelli, CEO of AAVantgarde..

“我们很高兴与世界级的CDMO AGC Biologics合作，他们在病毒载体制造方面拥有深厚的专业知识。这次合作标志着AAVantgarde的一个重要里程碑，因为我们继续在临床中推进我们创新的基因治疗管线，”AAVantgarde首席执行官Natalia Misciattelli表示。