ViGeneron GmbH, a next-generation gene therapy company, has announced a target-specific strategic collaboration and option agreement with Regeneron Pharmaceuticals Inc. (Regeneron) to develop and commercialize a gene therapy product based on ViGeneron’s novel engineered recombinant adeno-associated virus vectors (vgAAVs) to treat an inherited retinal disease (IRD).
Under the terms of the research collaboration, Regeneron and ViGeneron will create and validate vgAAV-based therapeutic candidates for one undisclosed IRD target. ViGeneron receives an upfront payment and research funding. Regeneron has an option for an exclusive license to develop, commercialize and manufacture the vgAAV-based product for the specific target. ViGeneron is eligible to receive an option exercise fee, development and commercial milestone payments, plus royalties on net sales, ViGeneron said in its press release.
ViGeneron’s vgAAV vector platform is designed to overcome the limitations of existing adeno-associated virus (AAV)-based gene therapies. To date, therapeutically impactful targeting of photoreceptors relies on subretinal vector delivery, which harbors substantial risks of retinal detachment and collateral damage, often without achieving widespread photoreceptor transduction. vgAAV vectors could potentially enable the efficient transduction of target cells via intravitreal injection that allows lateral spreading and minimizes the risk of retinal detachment caused by conventional subretinal injection, the company said.
“We are delighted to work with Regeneron to potentially provide an intravitreally delivered gene therapy for patients suffering from an inherited eye disease,” said Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron. “This agreement with Regeneron further validates the potential of our vgAAV platform, which is excellent for us and also delivers a deal value that contributes financing for our platform and proprietary program development activities. Furthermore, it fits into our strategy of developing proprietary programs for selected retinal targets through clinical trials, while maximizing our technology platforms for additional collaboration programs in retinal diseases, CNS and other disease areas with bellwether biopharma. Our aim is to overcome the current limitations of gene therapy and to bring a novel therapeutic approach to patients in need,” she added.