Chat with Filing

Ask questions about this document

Please log in or register to use the chat feature. Please register first.

RNS - ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease

Company: Vivoryon Therapeutics N.V. Date: 2025-06-05 Language: English Register to download

Loading document preview...

Document Preview Not Available

The file type (.html) cannot be displayed directly here.

Open Original File

Error loading document preview. Please try the 'Open Original File' link.

You're One Click Away From The Full Filing

Instantly access this document—and 2.8M more—for free. Select your preferred method:

Continue with Google Continue with Microsoft Continue with Facebook Continue with Amazon

 News Details 

Corporate | 5 June 2025 14:00

#   

ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease

VeonGen Therapeutics / Key word(s): Strategic Company Decision  

ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease  

  

05.06.2025 / 14:00 CET/CEST  

The issuer is solely responsible for the content of this announcement.  

  

- - -

**ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare  

Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease**

-   **VeonGen advances two proprietary, first-in-class gene therapies in the clinic, enabled by its novel vgRNA REVeRT and vgAAV technology platforms**
-   **Lead program VG801 receives FDA Rare Pediatric Disease Designation and is progressing in a first-in-human Phase 1/2 trial for Stargardt disease, with functional endpoint development underway through the FDA’s RDEA program**

**Munich, Germany, June 5, 2025** –– VeonGen Therapeutics, formerly known as ViGeneron, today announced its rebranding to reflect its evolution into a clinical-stage genetic medicine company focused on developing transformative gene therapies for patients with high unmet medical needs.

VeonGen has advanced two first-in-class gene therapy programs into clinical development, marking a significant step toward addressing diseases with no approved treatment options. These programs include VG801, a novel dual AAV gene therapy for Stargardt disease and other *ABCA4* \-related retinal disorders, and VG901, an intravitreally delivered AAV gene therapy for retinitis pigmentosa caused by *CNGA1* mutations. Both programs are enabled by VeonGen’s proprietary genetic medicine platforms, designed to address critical gene delivery challenges in AAV-based therapies: vgRNA REVeRT, which utilizes mRNA trans-splicing to deliver large genes exceeding the AAV cargo limit of 4.7 kb; and vgAAV, an engineered capsid platform optimized for safe and efficient delivery via intravitreal or subretinal administration.

VeonGen also announced that its lead program VG801 has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for the treatment of *ABCA4* mutation-associated retinal dystrophy. The investigational therapy is currently being evaluated in a first-in-human Phase 1/2 clinical trial, with patient dosing underway. Concurrently, VeonGen is developing a functional endpoint in collaboration with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program. VG801 aims to treat patients with biallelic *ABCA4* mutations linked to Stargardt disease and related retinal dystrophies – the most common inherited retinal disease, affecting about 1 in 8,000 to 10,000 people worldwide, and a leading cause of macular degeneration in children and young adults.

“This rebranding reflects our journey- from a platform innovator to a clinical-stage company with two gene therapies in the clinic. With VG801 and VG901 progressing in clinical trials and our platforms demonstrating robust translational potential, we are well positioned to expand the frontier of genetic medicine in ophthalmology and beyond,” said Dr. Caroline Man Xu, Co-founder & Chief Executive Officer 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.”

VeonGen’s proprietary gene therapy platforms are designed for broad therapeutic applicability. In addition to ophthalmology, the company is exploring expansion and partnerships in cardiovascular, central nervous system (CNS), and other disease areas.

**About VeonGen Therapeutics**

VeonGen Therapeutics is a clinical-stage genetic medicine company developing next-generation gene therapies for patients with high unmet medical needs. The company’s proprietary ophthalmology pipeline features two first-in-class clinical programs: VG801, a dual AAV gene therapy that leverages mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the full-length *ABCA4* gene for Stargardt disease and related retinal disorders; and VG901, an intravitreally delivered AAV gene therapy based on the vgAAV platform targeting retinitis pigmentosa caused by *CNGA1* mutations.

VeonGen’s three differentiated technology platforms serve as a robust toolbox designed to address critical gene delivery challenges and unmet needs associated with AAV-based therapies. The vgAAV platform features engineered AAV capsids with enhanced transduction efficiency and the ability to overcome biological barriers, enabling less invasive delivery routes such as intravitreal and systemic administration. The vgRNA REVeRT (Reconstitution via mRNA Trans-splicing) platform enables delivery and reconstitution of large genes—exceeding the 4.7 kb AAV cargo limit—at the mRNA level in tissues targeted by a selected AAV capsid. The third platform, AAV Transactivation, leverages CRISPR/Cas-mediated transcriptional regulation to enable in vivo activation or repression of disease-relevant genes—extending the potential of AAV-based therapies beyond gene supplementation and enabling novel applications in genetic diagnostics.

With its strong potential for applications beyond ophthalmology, VeonGen is also exploring expansion and partnerships in cardiovascular, central nervous system (CNS), and other disease areas. The company was founded in 2017 by a team with deep expertise in AAV vector engineering, gene therapy development, and clinical translation. VeonGen is headquartered in Munich, Germany.

For more information, please visit [www.veongen.com](https://eqs-cockpit.com/cgi-bin/fncls.ssp?fn=redirect&url=0e76ff059e9469076bc750ee69740a78&application_id=2151410&site_id=airport_aws~~~7cc68db1-74de-43c8-af69-2ae0a97efdc3&application_name=news)

|     |     |
| --- | --- |
| **VeonGen Therapeutics Contact**  <br><br>Dr. Caroline Man Xu  <br><br>Co-Founder and CEO  <br><br>[info@veongen.com](mailto:info@veongen.com) | **VeonGen Therapeutics Media Contact**  <br><br>MC Services AG  <br><br>Shaun Brown / Julia von Hummel  <br><br>phone: +49 (0)89 2102280  <br><br>[veongen@mc-services.eu](mailto:veongen@mc-services.eu) |

  

- - -

05.06.2025 CET/CEST Dissemination of a Corporate News, transmitted by EQS News – a service of EQS Group.  

The issuer is solely responsible for the content of this announcement.  

  

The EQS Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases.  

Archive at www.eqs-news.com  

- - -

2151410  05.06.2025 CET/CEST

![](https://eqs-cockpit.com/cgi-bin/fncls.ssp?fn=show_t_gif&application_id=2151410&application_name=news&site_id=airport_aws~~~7cc68db1-74de-43c8-af69-2ae0a97efdc3)