KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treatments, which have demonstrated improvement in vision and reduction in fluid. A one-time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.
In the past few years, an increasing volume of mature data has been obtained from clinical trials in the fields of genome editing and gene therapy, demonstrating the efficacy and safety of gene therapy for a range of severe diseases in humans.
Gene therapy is an emerging treatment approach which is designed to introduce foreign genetic material into target cells, to correct or compensate for genetic defects and abnormalities to treat diseases.
The gene therapy product KH631 has shown sustained efficacy in preclinical disease models and KH631 is currently approved to begin investigations in human clinical trials.