SHANGHAI

上海

,

，

May 10, 2025

2025年5月10日

/PRNewswire/ -- Skyline Therapeutics, an innovation-driven

/PRNewswire/ -- Skyline Therapeutics，一家创新驱动的

clinical-stage

临床阶段

gene therapy company focused on developing unique and novel solutions for rare and severe diseases, today announced a late-breaking abstract presentation on

专注于开发罕见和严重疾病独特和新颖解决方案的基因治疗公司，今天宣布了一份关于延迟发布的摘要展示。

SKG1108

SKG1108

, its investigational optogenetic gene therapy for

其研究性的光遗传学基因疗法用于

Retinitis Pigmentosa (RP)

视网膜色素变性 (RP)

, at the

， 在

American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting

美国基因与细胞治疗学会（ASGCT）第28届年会

(May 13–17, 2025,

（2025年5月13日至17日，

New Orleans, Louisiana

路易斯安那州新奥尔良

).

).

ASGCT 2025 Late-Breaking Abstract

ASGCT 2025 晚期摘要征集

Abstract Title: SKG1108: Optogenetic Gene Therapy with AAV-BWLP for Visual Restoration in Retinitis Pigmentosa via a Single Intravitreal Injection

摘要标题：SKG1108：通过单次玻璃体内注射AAV-BWLP进行视网膜色素变性的光遗传基因治疗以恢复视力

Abstract No.：2023

摘要编号：2023

Date & Time:

日期和时间：

May 13, 2025

2025年5月13日

|

|

6:00 PM

下午6:00

–

–

7:30 PM CT

晚上7点30分（中部时间）

About SKG1108

关于SKG1108

SKG1108 is an

SKG1108 是一个

optogenetic adeno-associated virus (AAV) gene therapy

光遗传学腺相关病毒（AAV）基因治疗

vector designed to restore vision in RP patients with photoreceptor degeneration. Its novel design combines a proprietary

旨在恢复视网膜色素变性（RP）患者视力的载体，其创新设计结合了一种专利技术

intravitreal capsid

玻璃体内衣壳

and a uniquely engineered

并且独特设计的

Broad Wavelength Light-sensing Protein

广波长光敏蛋白

(BWLP)

（BWLP）

comprising opsins of multiple sources, which demonstrated

包含来自多个来源的视蛋白，这表明

strong and broad wave-length range light-responsive activity

强而宽波长范围的光响应活性

in

在

in vitro

体外

electrophysiological studies. In

电生理学研究。在

in vivo

体内

studies, a

研究，一个

single intravitreal injection

单次玻璃体内注射

of SKG1108 showed

SKG1108的数据显示

superior efficacy and safety

优越的疗效和安全性

in

在

rd1

rd1

mice, with striking functional improvements in visual pathway electrophysiology, light perception and visual acuity behavioral tests. The therapy delivers a single-stranded DNA payload encoding light-activatable proteins to the retina, bypassing the need to target specific genetic mutations. By generating new photo-sensing cells, SKG1108 aims to .

小鼠的视觉通路电生理、光感知和视力行为测试均有显著的功能改善。该疗法将编码光激活蛋白的单链DNA递送到视网膜，绕过了针对特定基因突变的需求。通过生成新的感光细胞，SKG1108旨在实现。

restore visual function

恢复视觉功能

in late-stage RP patients, regardless of their underlying genetic defect. SKG1108 received

在晚期RP患者中，无论其潜在的基因缺陷如何。SKG1108已获得

U.S. FDA Orphan Drug Designation (ODD)

美国FDA孤儿药资格认定（ODD）

in

在

September 2024

2024年9月

.

。

About Retinitis Pigmentosa (RP)

关于视网膜色素变性（RP）

Retinitis Pigmentosa (RP) is a type of inherited retinal diseases (IRDs) linked to mutations in over 100 different genes, leading to progressive vision loss that typically begins with night blindness (rod degeneration), followed by tunnel vision and eventual legal blindness due to near-total photoreceptor death (rod and cone degeneration).

视网膜色素变性（RP）是一种遗传性视网膜疾病（IRD），与超过100种不同基因的突变相关，导致进行性视力丧失，通常始于夜盲症（视杆细胞退化），随后出现管状视野，并最终因几乎全部光感受器死亡（视杆细胞和视锥细胞退化）而导致法定失明。

Currently, no effective treatments worldwide can effectively slow, halt, or reverse photoreceptor degeneration, and existing gene-specific therapies only apply to a small subset of RP patients. SKG1108's mutation-agnostic optogenetic approach—designed to restore vision independent of genetic defects—has the potential to become a transformative treatment for the vast majority of RP patients, including those with advanced disease..

目前，全球范围内尚无有效的治疗方法可以有效减缓、阻止或逆转光感受器的退化，而现有的基因特异性疗法仅适用于一小部分视网膜色素变性（RP）患者。SKG1108的与突变无关的光遗传学方法——旨在不依赖于基因缺陷的情况下恢复视力——有潜力成为大多数RP患者的革命性治疗手段，包括那些病情已进入晚期的患者。

About Skyline Therapeutics

关于天际线治疗公司

Skyline Therapeutics is an innovation-driven, clinical-stage gene therapy company focused on developing unique and novel therapeutic solutions for rare and severe diseases with high unmet needs. Leveraging our cutting-edge adeno-associated virus (AAV) platform, which encompasses multiple proprietary technologies for capsid discovery, vector design and engineering, process development and GMP manufacturing, we are advancing a diverse pipeline of gene therapies targeting ocular, neurological, and cardiovascular disorders.

天际线治疗公司是一家创新驱动的临床阶段基因治疗公司，专注于为高度未满足需求的罕见和严重疾病开发独特且新颖的治疗方案。依托我们尖端的腺相关病毒（AAV）平台，该平台包含多种用于衣壳发现、载体设计与工程、工艺开发和GMP生产的专有技术，我们正在推进针对眼科、神经科和心血管疾病的多样化基因治疗管线。

Our lead programs are rapidly progressing through clinical trials with regulatory approvals and special designations from the US FDA and China NMPA, bringing us closer to providing life-changing therapies to patients in need. .

我们的主导项目正在快速通过临床试验，并获得了美国FDA和中国NMPA的监管批准和特别指定，使我们更接近为有需要的患者提供改变生命的治疗方案。

www.skytx.com

www.skytx.com

About ASGCT

关于ASGCT

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of gene, related cell and nucleic acid therapies, as well as promotion of professional and public education in the field. With more than 5,000 members in the US and worldwide, ASGCT is the largest association of individuals involved in gene and cell therapy research.

美国基因与细胞治疗学会（ASGCT）是一个专业的非营利性医学和科学组织，致力于基因、相关细胞和核酸疗法的理解、开发与应用，并推动该领域的专业和公众教育。ASGCT拥有5000多名美国及全球会员，是参与基因和细胞治疗研究的最大个人协会。

The ASGCT Annual Meeting is the premier event for gene and cell therapy professionals to learn from the latest scientific research and stay current on new technologies..

ASGCT年会是基因和细胞治疗专业人士学习最新科学研究和掌握新技术的首要活动。

SOURCE Skyline Therapeutics

来源：天际线治疗公司

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