Atsena Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational gene therapy, ATSN-201, for the treatment of X-linked retinoschisis (XLRS).

Atsena Therapeutics 宣布，美国食品药物管理局 (FDA) 已授予其在研基因疗法 ATSN-201 再生医学先进疗法 (RMAT) 资格，用于治疗 X 连锁视网膜劈裂症 (XLRS)。

About ATSN-201 and AAV.SPR Technology

关于ATSN-201和AAV.SPR技术

ATSN-201 is a gene therapy candidate that utilizes AAV.SPR, Atsena’s proprietary spreading capsid technology. This platform is designed to deliver therapeutic levels of gene expression specifically to photoreceptors in the central retina, without introducing the surgical risks associated with foveal detachment.

ATSN-201 是一种基因疗法候选药物，利用了 Atsena 专有的扩散衣壳技术 AAV.SPR。该平台旨在特异性地将治疗水平的基因表达递送至视网膜中央的光感受器，同时避免与黄斑脱离相关的手术风险。

The therapy targets mutations in the RS1 gene, the underlying cause of XLRS..

该疗法针对 RS1 基因突变，这是 XLRS 的根本原因。

RMAT Designation: Accelerating Development of Regenerative Therapies

RMAT指定：加速再生疗法的开发

The RMAT designation, established under the 21st Century Cures Act, is granted to regenerative medicine therapies intended to treat, modify, reverse, or cure serious or life-threatening conditions. To qualify, a therapy must also show preliminary clinical evidence of potential benefit in addressing an unmet medical need..

RMAT 指定是根据《21世纪治愈法案》设立的，授予用于治疗、改善、逆转或治愈严重或危及生命状况的再生医学疗法。要获得资格，一种疗法还必须显示出应对未满足医疗需求的初步临床证据。

RMAT status offers several regulatory advantages, including:

RMAT状态提供若干监管优势，包括：

• Early and frequent FDA guidance on drug development

• FDA对药物开发的早期和频繁指导

• The possibility of using surrogate or intermediate endpoints

• 使用替代或中间终点的可能性

• Options for accelerated approval pathways

• 加速审批途径的选择

• Potential for priority review of a future Biologics License Application (BLA)

• 未来生物制品许可申请（BLA）优先审查的潜力

CEO Statement: Advancing Treatments for Inherited Retinal Diseases

首席执行官声明：推进遗传性视网膜疾病的治疗

“We’re honored that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201, further underscoring its potential to address the urgent, unmet need in XLRS—a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics..

“我们感到荣幸的是，美国食品药品监督管理局（FDA）已授予ATSN-201再生医学先进疗法（RMAT）资格，这进一步突显了其在解决XLRS（一种罕见的遗传性视网膜疾病，目前尚无获批疗法）中迫切未满足需求的潜力，”Atsena Therapeutics首席执行官Patrick Ritschel表示。

“This regulatory momentum, coupled with the recent close of our oversubscribed $150 million Series C financing, reinforces our commitment to advancing meaningful gene therapies that have the potential to improve vision and quality of life for individuals living with XLRS and other inherited retinal diseases.”.

“这种监管势头，加上我们最近超额认购的 1.5 亿美元 C 轮融资的完成，进一步坚定了我们推进有意义的基因疗法的决心，这些疗法有潜力改善 XLRS 和其他遗传性视网膜疾病患者的生活质量和视力。”

Ongoing Clinical Trial: The LIGHTHOUSE Study

正在进行的临床试验：LIGHTHOUSE 研究

The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase 1/2, dose-escalation and dose-expansion trial. The study involves male patients aged 6 and older diagnosed with XLRS due to RS1 gene mutations.

LIGHTHOUSE 研究正在评估 ATSN-201 的安全性和耐受性，这是一项 1/2 期剂量递增和剂量扩展试验。该研究涉及年龄在 6 岁及以上的男性患者，他们因 RS1 基因突变而被诊断为 XLRS。

Unmet Needs in X-Linked Retinoschisis

X连锁视网膜劈裂症的未满足需求

XLRS is a rare, inherited retinal disease typically diagnosed in early childhood. It affects approximately 30,000 males across the United States and the European Union, and currently, no approved treatments are available.

XLRS是一种罕见的遗传性视网膜疾病，通常在儿童早期被诊断出来。它影响美国和欧盟大约3万名男性，目前尚无获批的治疗方法。