ViGeneron Rebrands as VeonGen, Earns FDA RPDD for Gene Therapy

ViGeneron更名为VeonGen，其基因疗法获FDA授予RPDD资格

June 16, 2025

2025年6月16日

VeonGen Therapeutics has officially announced its rebranding from ViGeneron, marking a strategic transformation into a clinical-stage genetic medicine company dedicated to developing innovative gene therapies for diseases with high unmet medical needs.

VeonGen Therapeutics正式宣布从ViGeneron更名，标志着其战略转型为一家临床阶段的基因药物公司，致力于为具有高度未满足医疗需求的疾病开发创新基因疗法。

Transition to a Clinical-Stage Genetic Medicine Company

转型为临床阶段的基因医药公司

The rebranding reflects VeonGen’s evolution from a platform innovator to a company with active clinical programs. It has advanced two first-in-class gene therapy candidates into human trials:

此次品牌重塑反映了VeonGen从平台创新者到拥有活跃临床项目的公司的演变。该公司已将两款首创基因治疗候选药物推进到人体试验阶段：

• VG801:

• VG801:

A dual adeno-associated virus (AAV) gene therapy targeting Stargardt disease and other ABCA4-related retinal disorders.

一种针对斯塔加特病和其他ABCA4相关视网膜疾病的双重腺相关病毒（AAV）基因疗法。

• VG901:

• VG901:

An AAV-based therapy designed for intravitreal delivery to treat retinitis pigmentosa caused by CNGA1 mutations.

一种基于AAV的疗法，设计用于玻璃体内递送，以治疗由CNGA1基因突变引起的视网膜色素变性。

Proprietary Platforms Supporting Gene Therapy Development

支持基因治疗开发的专有平台

Both therapies are supported by VeonGen’s proprietary platforms that address the limitations of conventional AAV-based gene delivery:

这两种疗法都得到了VeonGen专有平台的支持，该平台解决了传统基于AAV的基因递送的局限性：

• vgRNA REVeRT:

• vgRNA REVeRT:

Uses mRNA trans-splicing to enable delivery of large genes exceeding the AAV’s 4.7 kb cargo limit.

利用mRNA反式剪接技术，实现超过AAV 4.7 kb载荷限制的大基因递送。

• vgAAV:

• vgAAV:

A capsid-engineering platform optimized for safe, efficient gene delivery via intravitreal or subretinal routes.

一种优化的衣壳工程平台，用于通过玻璃体内或视网膜下途径进行安全、高效的基因递送。

FDA Rare Pediatric Disease Designation for VG801

FDA罕见儿科疾病认定VG801

VG801, VeonGen’s lead investigational product, has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for the treatment of ABCA4 mutation-associated retinal dystrophy. This novel gene therapy is now being evaluated in a first-in-human Phase 1/2 clinical trial, with patient dosing already in progress..

VG801，VeonGen公司的主要研究产品，已被美国食品药品监督管理局（FDA）授予治疗ABCA4突变相关视网膜营养不良的罕见儿科疾病认定（RPDD）。这种新型基因疗法目前正在进行首次人体 Phase 1/2 临床试验，患者给药已在进行中。

Simultaneously, VeonGen is collaborating with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program to develop a functional clinical endpoint. VG801 is aimed at treating patients with biallelic ABCA4 mutations associated with Stargardt disease and related inherited retinal dystrophies..

同时，VeonGen正通过罕见病终点推进（RDEA）试点计划与FDA合作，开发一种功能性临床终点。VG801旨在治疗与斯塔加特病及相关遗传性视网膜营养不良相关的双等位ABCA4基因突变患者。

Strategic Vision and Future Expansion

战略愿景与未来扩展

“This rebranding reflects our journey—from a platform innovator to a clinical-stage company with two gene therapies in the clinic,” said Dr. Caroline Man Xu, Co-founder & CEO of VeonGen Therapeutics. “The FDA Rare Pediatric Disease Designation for VG801 not only highlights the strength of our scientific approach but also reinforces our focus on accelerating the development and delivery of transformative therapies for patients in urgent need.”.

“此次品牌重塑反映了我们的历程——从一个平台创新者发展为一家拥有两种基因疗法进入临床阶段的公司，”VeonGen Therapeutics联合创始人兼首席执行官徐曼博士表示。“VG801获得FDA罕见儿科疾病认定不仅彰显了我们科学方法的实力，也进一步强化了我们专注于加速为急需治疗的患者开发并交付变革性疗法的决心。”

Beyond ophthalmology, VeonGen is also exploring therapeutic applications and strategic partnerships in cardiovascular, central nervous system (CNS), and other high-impact disease areas.

除了眼科领域，VeonGen 还在心血管、中枢神经系统（CNS）及其他高影响力疾病领域探索治疗应用和战略合作伙伴关系。