
Gene Therapy Developer
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According to Vascular Network News, recently, ViGeneron completed its A+ round of financing, led by Huagai Capital with participation from well-known industry funds. The funds from this round will mainly be used to advance the clinical development of two ophthalmic gene therapy drugs.
ViGeneron, a cutting-edge gene therapy company based in Munich, Germany, is dedicated to addressing unmet needs in the field of ophthalmic disease treatment and has built a diversified product portfolio. Among these, two products have gained recognition from the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) and have successfully advanced to the clinical trial stage.

ViGeneron Pipeline
In a recently completed financing activity, ViGeneron announced that its gene therapy VG801 has been approved by the U.S. FDA to enter the IND stage of Phase 1/2 clinical trials. As the world’s first gene therapy utilizing mRNA trans-splicing recombination technology, VG801 aims to treat Stargardt disease and retinal dystrophy associated with ABCA4 gene mutations. The therapy leverages ViGeneron's proprietary dual adeno-associated virus (AAV) vector REVeRT technology, successfully addressing the challenge of packaging the large sequence of the ABCA4 gene (approximately 6.7 kb) into a single AAV vector. REVeRT employs two AAV vectors, each carrying half of the ABCA4 coding sequence, generating two precursor mRNAs that undergo trans-splicing to form a complete mRNA, which is then translated into a functional protein. Additionally, VG801 incorporates a novel capsid generated from ViGeneron's proprietary vgAAV technology platform to achieve broad retinal transduction. Stargardt disease, the most common inherited retinal disease (IRD), affects approximately 1 in every 8,000 to 10,000 individuals worldwide. Currently, there are no approved therapies to halt its progression, highlighting the urgent need for effective treatments. Recently, clinical trials for VG801 have also been initiated in China, bringing a ray of hope to patients.
At the same time, ViGeneron announced that its proposal has been selected by the U.S. FDA to be included in the Rare Disease Endpoint Advancement (RDEA) pilot program. This program aims to promote innovation and development in drug development projects for rare diseases. As a member of the RDEA program, ViGeneron will work closely with the FDA to explore the development of new efficacy endpoints and have more opportunities to communicate with the FDA. Notably, between 2024 and 2027, the FDA will only accept three RDEA projects per year.
Dr. Caroline Man Xu, co-founder and CEO of ViGeneron, stated: "We sincerely thank Huagai Capital for their trust and support. This financing will significantly advance the global development of our two clinical programs and the progress of our three technology platforms. ViGeneron will continue to focus on leading the research and development of gene therapies through innovative technologies, deepen our expertise in ophthalmic gene therapy, overcome the limitations of current gene therapies, and provide patients with better treatment options."
Wang Xun, Chief Investment Officer of Huagai Healthcare Fund, stated: "The potential of AAV in drug development has been proven, and the eye is undoubtedly an ideal field for gene therapy breakthroughs, with a significant unmet clinical need. ViGeneron's international team has years of deep expertise in the gene therapy field, with extensive experience from laboratory to clinical translation. The company has overcome the limitations of existing vectors through multiple technology platforms, earning recognition from several multinational pharmaceutical companies. In particular, the recent FDA IND approval for VG801 marks an important milestone in the company’s development and a major 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 internationally leading innovative gene therapy products, bringing light to patients."
Source: Arterial Network;ViGeneron Official Website
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