VeonGen Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for VG801 Gene Therapy for Stargardt Disease

MUNICH, Aug. 21, 2025 (GLOBE NEWSWIRE) -- VeonGen Therapeutics, a clinical-stage genetic medicine company developing next-generation gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to VG801, its lead investigational gene therapy for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies. By delivering a full-length, functional ABCA4 gene, VG801 has the potential to address the underlying genetic root cause of Stargardt disease across all ABCA4 mutations.

RMAT is a designation granted by the FDA to regenerative medicine therapies, including gene therapies, that are being developed to treat, modify, reverse, or cure serious or life-threatening conditions. To qualify, an investigational product must show preliminary clinical evidence indicating potential to address unmet medical needs for such diseases or conditions. RMAT designation provides enhanced engagement with the FDA, opportunities for more streamlined clinical and manufacturing development, potential priority review of a biologics license application (BLA), and other pathways to expedite development and review.

With this milestone, VG801 now holds RMAT, Rare Pediatric Disease, and Orphan Drug designations. VeonGen has also been selected to collaborate with the FDA under the Rare Disease Endpoint Advancement (RDEA) pilot program to develop a novel functional endpoint for Stargardt disease.

“Receiving RMAT designation is strong recognition of VG801’s therapeutic potential for Stargardt disease, the most common inherited retinal disorder with no approved therapies,” said Dr. Caroline Man Xu, Co-founder & Chief Executive Officer of VeonGen Therapeutics. “This recognition highlights the promise of our novel vgRNA REVeRT and vgAAV platforms and provides an opportunity to accelerate VG801’s development and deliver a much-needed therapy to patients as quickly and efficiently as possible.”

About VG801

VG801 is a dual-AAV gene therapy in development for Stargardt disease and other ABCA4-associated retinal dystrophies. It is designed to restore full-length, functional ABCA4 expression through VeonGen’s proprietary vgRNA REVeRT platform, combined with engineered vgAAV capsids that enable efficient delivery to photoreceptor cells. By delivering a functional ABCA4 gene, VG801 has the potential to address the underlying genetic cause of all forms of ABCA4-mutated Stargardt disease. VG801 is currently being evaluated in a Phase I/II clinical trial, with patient dosing underway.

About Stargardt Disease

Stargardt disease is the most prevalent inherited retinal disorder, affecting approximately 1 in 8,000–10,000 individuals worldwide. Caused by mutations in the ABCA4 gene, Stargardt disease is characterized by progressive central vision loss that often begins in childhood or adolescence, leading to a gradual decline in visual acuity and, in some cases, legal blindness. Currently, there are no approved treatments for Stargardt disease, highlighting a critical unmet medical need.

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 ophthalmology pipeline includes VG801, a dual-AAV gene therapy for Stargardt disease and ABCA4-related retinal disorders, and VG901, an intravitreally delivered AAV gene therapy for retinitis pigmentosa caused by CNGA1 mutations.

VeonGen’s proprietary platforms vgAAV, vgRNA REVeRT, and AAV Transactivation serve as a robust toolbox designed to overcome key challenges in gene therapy by enabling efficient gene delivery, reconstitution of large genes beyond traditional AAV limits, and transcriptional regulation of disease-relevant genes, thereby expanding the potential of AAV-based therapies. Headquartered in Munich, Germany, VeonGen is also exploring applications and partnerships in cardiovascular, central nervous system (CNS), and other disease areas.

For more information, please visit www.veongen.com.

Legal Disclaimer:

EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.