ViGeneron Secures Series A+ Financing Led by China Renaissance Capital and Receives FDA Clearance for VG801, the World’s First mRNA Trans-Splicing Gene Therapy for Stargardt Disease

Recently, ViGeneron completed its A+ round of financing, led by Huagai Capital with participation from well-known industry funds. The proceeds from this round will mainly be used to advance the clinical development of two ophthalmic gene therapy drugs.

ViGeneron is an innovative gene therapy company headquartered in Munich, Germany. It has developed a robust product pipeline targeting unmet needs in ophthalmic diseases, with two of its products having received approval from the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) and entering the clinical trial stage.

Upon completing this round of financing, the company announced that the IND application for the Phase 1/2 clinical trial of its investigational gene therapy VG801 has been approved by the U.S. FDA. This marks the world's first mRNA trans-splicing recombination gene therapy to be approved by the FDA for clinical entry, targeting Stargardt disease and other retinal dystrophies associated with ABCA4 gene mutations. VG801 utilizes ViGeneron's proprietary dual adeno-associated virus (AAV) vector REVeRT (Recombination through mRNA trans-splicing) technology, addressing the challenge posed by the large size of the DNA sequence encoding human ABCA4 (approximately 6.7 kb), which cannot fit into a single AAV vector. REVeRT consists of two AAV vectors, each carrying half of the ABCA4 coding sequence, producing two separate pre-mRNA molecules. These pre-mRNA molecules undergo trans-splicing to form a complete mRNA, which is then translated into a functional protein. VG801 also employs a novel vgAAV capsid generated based on ViGeneron’s proprietary vgAAV technology platform, designed to achieve widespread retinal transduction. Stargardt disease is the most common inherited retinal disease (IRD), affecting approximately one in every 8,000 to 10,000 people worldwide. Currently, there are no approved treatments available to halt the progression of the disease, highlighting the urgent unmet medical need for effective therapies. Clinical trials for VG801 have also recently commenced in China, offering hope to many patients.

ViGeneron Also Announces FDA Selection of Proposal for Rare Disease Endpoint Advancement (RDEA) Pilot Program. The RDEA program aims to promote innovation and advance the development of rare disease drug programs. As part of the RDEA program, ViGeneron will work closely with the FDA during the development of new efficacy endpoints and gain increased opportunities for meetings with the FDA. Notably, from 2024 to 2027, the FDA will accept a maximum of three RDEA projects per year.

Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron"We are very grateful for the trust and support from Huagai Capital. This financing will greatly promote the company’s global development of two clinical projects and the development of three technology platforms. ViGeneron will continue to advance the research and development of gene therapies through innovative technologies, focusing on the field of ophthalmic gene therapy, breaking through the current limitations of gene therapy, and bringing better treatment options to patients in need."

Wang Xun, Chief Investment Officer of Huagai Healthcare Fund"AAV's druggability has been validated, and the eye is an ideal field for breakthroughs in gene therapy, with significant unmet clinical needs. ViGeneron’s international team has deep expertise in gene therapy, with decades of experience in translating gene therapy 'from bench to bedside.' The company’s multiple technology platforms overcome limitations of existing vectors and have gained recognition from several multinational corporations (MNCs). Two of its products have entered clinical stages, especially VG801, which recently received FDA IND approval—a key milestone in the company’s development and a breakthrough in the treatment of Stargardt disease. We are delighted to lead this round of financing and look forward to the successful development of ViGeneron’s world-leading innovative gene therapy products, bringing light to patients."

ViGeneron was founded in 2017, with its headquarters located in Munich, Germany. It is committed to bringing innovative gene therapies to patients in need. ViGeneron's three next-generation gene therapy technology platforms aim to overcome the limitations of existing AAV (adeno-associated virus)-based gene therapies. The first is the vgAAV vector platform, which achieves more efficient and innovative delivery methods, such as intravitreal injection and systemic administration, by optimizing the transduction efficiency of viral vectors. The second is the REVeRT (Recombination via mRNA Trans-Splicing) technology platform, capable of effectively recombining large genes (>5kb) in any specific capsid-targeted tissue, offering new possibilities for the treatment of large genes. Third, AAV transactivation is a CRISPR-Cas-based AAV gene therapy platform that can simultaneously enhance or suppress the expression of one or more genes.