VG901 is the only clinical-stage therapy designed to deliver the functional CNGA1 gene to retinal photoreceptor target cells in retinitis pigmentosa patients with mutations in the CNGA1 gene.VG901 uses ViGeneron’s proprietary next-generation vgAAV platform and is injected intravitreally (IVT), offering less invasive administration, broader vector distribution, and reduced risk of retinal damage linked with other methods of administration.The FDA has granted VG901 Orphan Drug Designation.MUNICH, Germany, April 10, 2024 — ViGeneron GmbH, a next-generation clinical stage gene therapy company, announced that the first patient has been dosed in its Phase Ib clinical trial to evaluate intravitreal injection of VG901 to treat retinitis pigmentosa (RP). This milestone marks significant progress in the company’s use of its next-generation technology platforms to develop gene therapies for critical unmet medical needs. “Delivering the functional CNGA1 gene to retinal photoreceptor target cells, VG901 offers a therapeutic potential that addresses the underlying genetic cause for retinitis pigmentosa patients,” commented Prof. Dr. Katarina Stingl, Head of the Clinic for Hereditary Retinal Degenerations in the Center for Ophthalmology and the Center for Rare Eye Diseases at the University of Tübingen, Germany, and the Principal Investigator for this trial. “We are excited to learn about this novel therapy and hope to make a meaningful difference to patients’ lives.” “Dosing our first patient in the VG901 Phase Ib clinical trial is a significant step forward for the company and for the patients we aim to benefit. VG901 has also been granted FDA Orphan Drug Designation status. We look forward to progressing the clinical development of this potentially transformative therapy,” said Dr. Caroline Man Xu, ViGeneron’s Co-founder and CEO. “Not only is the Phase Ib trial designed to provide key insights into the safety and preliminary efficacy of VG901, it is also a pivotal step in validating our next generation vector platform vgAAV, which has demonstrated superior transduction efficiency and enables intravitreal delivery.” The on-going Phase Ib clinical trial is an open-label, single-arm, dose-escalation study investigating the safety, tolerability, and preliminary efficacy of an intravitreal injection of VG901, a first-in-class CNGA1 gene therapy for autosomal recessive RP. For more information on the trial, please visit clinicaltrial.gov []. About Retinitis Pigmentosa (RP) Retinitis pigmentosa (RP) is a group of related eye disorders causing progressive vision loss. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision”, central visual impairment, reduced visual acuity, and ultimately, blindness. Retinitis pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively. Mutations in the CNGA1 gene, encoding subunits of CNG channels in rod photoreceptors, are reported to cause approximately 2% – 8% of autosomal recessive retinitis pigmentosa (arRP). About ViGeneron GmbH ViGeneron is dedicated to bringing gene therapy innovations to those in need. The company is developing its proprietary, clinical-stage gene therapy pipeline to treat ophthalmic diseases and collaborating with leading biopharmaceutical players in retinal diseases, CNS, cardiovascular, and other disease areas. ViGeneron’s three novel next-generation gene therapy platforms are designed to overcome the limitations of existing adeno-associated virus (AAV)-based gene therapies. The vgAAV vector platform enables superior transduction efficiency of target cells and is designed to overcome biological barriers, enabling less invasive routes of administration such as intravitreal and systemic administration. The REVeRT (REconstitution Via mRNA Trans-splicing) technology platform allows for the efficient reconstitution of large genes (>5kb) in any tissue targetable with a given capsid. The AAV Transactivation is a CRISPR-Cas–based AAV gene therapy platform that regulates one or multiple genes in vivo by increasing or inhibiting their expression. Privately-owned ViGeneron was founded in 2017 by a team with experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit . ViGeneron ContactViGeneron GmbHDr. Caroline Man Xu Co-Founder and CEOViGeneron Media ContactMC Services AGShaun Brown / Julia von Hummelphone: +49 (0)89 2102280