FDA Clears IND for Opus Genetics’ OPGx-BEST1 Gene Therapy

FDA批准了Opus Genetics的OPGx-BEST1基因疗法的IND申请

August 20, 2025

2025年8月20日

Opus Genetics has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for OPGx-BEST1, its gene therapy candidate for the treatment of BEST1-related inherited retinal disease (IRD). This regulatory milestone paves the way for the initiation of a Phase 1/2 clinical trial in the second half of 2025..

Opus Genetics 已获得美国食品药品监督管理局（FDA）对其用于治疗BEST1相关遗传性视网膜疾病（IRD）的基因疗法候选药物 OPGx-BEST1 的新药临床试验（IND）许可。这一监管里程碑为在 2025 年下半年启动 1/2 期临床试验铺平了道路。

Addressing an Untreated Retinal Condition

治疗未经处理的视网膜疾病

BEST1-related IRD, also known as Best disease or vitelliform macular dystrophy, is a rare inherited retinal disorder caused by mutations in the BEST1 gene. These mutations result in progressive macular degeneration, often leading to significant vision loss or blindness. Clinical features include retinal lesions and symptoms such as dimness of vision, metamorphopsia, and scotoma..

BEST1相关的IRD，也被称为Best病或卵黄样黄斑营养不良，是一种由BEST1基因突变引起的罕见遗传性视网膜疾病。这些突变导致进行性的黄斑变性，常引发显著的视力丧失或失明。临床特征包括视网膜病变以及视力模糊、视物变形和盲点等症状。

The disease mechanism involves disruption of the BEST1 channel, a calcium-activated chloride channel critical for ion transport within retinal pigment epithelium (RPE) cells. When functioning properly, this channel helps maintain homeostasis between rod and cone photoreceptors and the RPE. Mutations in the BEST1 gene impair this homeostatic balance, causing degradation of the interphotoreceptor matrix (cIPM) and the microvilli (cMV) connections with the RPE.

该疾病的机制涉及BEST1通道的破坏，这是一种对视网膜色素上皮（RPE）细胞内离子转运至关重要的钙激活氯通道。当其功能正常时，该通道有助于维持视杆和视锥光感受器与RPE之间的稳态。BEST1基因的突变会损害这种稳态平衡，导致光感受器间基质（cIPM）和微绒毛（cMV）与RPE连接的退化。

As a result, vitelliform lesions form between the RPE and Bruch’s membrane (BM)/choroid (CH), eventually leading to RPE atrophy and photoreceptor cell death, resulting in progressive vision loss..

因此，卵黄样病变在视网膜色素上皮（RPE）与布鲁赫膜（BM）/脉络膜（CH）之间形成，最终导致视网膜色素上皮萎缩和光感受器细胞死亡，从而引起进行性视力丧失。

A Gene Therapy Approach to Restoring Function

基因疗法恢复功能的方法

OPGx-BEST1 is developed using Opus Genetics’ proprietary adeno-associated virus (AAV)-based gene therapy platform. The therapy is engineered to deliver a functional copy of the BEST1 gene directly into the RPE cells, targeting the root cause of the disease by replacing the faulty gene.

OPGx-BEST1是利用Opus Genetics公司专有的基于腺相关病毒（AAV）的基因治疗平台开发的。该疗法通过将功能正常的BEST1基因直接递送到视网膜色素上皮（RPE）细胞中，旨在通过替换缺陷基因来针对疾病的根源。

The upcoming Phase 1/2 clinical trial will be a multi-center, open-label study evaluating the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1. The study will enroll patients with genetically confirmed BEST1-related IRD, and initial data is expected in the first quarter of 2026..

即将进行的 1/2 期临床试验将是一项多中心、开放标签的研究，评估单次视网膜下注射 OPGx-BEST1 的安全性、耐受性和初步疗效。该研究将招募基因确认的与 BEST1 相关的 IRD 患者，预计将在 2026 年第一季度获得初始数据。

Commenting on the IND clearance, George Magrath, MD, CEO of Opus Genetics, stated:

就IND clearance，Opus Genetics首席执行官George Magrath博士表示：

“BEST1-related IRDs have no approved treatments today, leaving patients and families with uncertainty about the future of their vision. The OPGx-BEST1 trial will be our third ongoing clinical program, reflecting the depth of our pipeline and our commitment to advancing multiple therapies in parallel for patients with urgent, unmet needs.”.

“与BEST1相关的IRD目前尚无获批的治疗方法，这让患者及其家人对未来的视力状况充满不确定性。OPGx-BEST1试验将成为我们第三个正在进行的临床项目，反映出我们研发管线的深度以及我们致力于为有迫切需求且未满足的患者推进多种疗法并行发展的承诺。”