Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, and the TSC Alliance recently announced a collaboration to advance gene therapy programs designed to treat tuberous sclerosis complex (TSC), a rare genetic disease. Gene therapy programs advanced through the collaboration will use Apertura’s TfR1 CapX™, a novel, intravenously delivered AAV capsid designed to target human transferrin receptor 1 (hTfR1), cross the blood-brain barrier, and enable broad distribution to the brain and spinal cord.
Statement from the TSC Alliance: We are excited about this new partnership to help advance gene therapy for TSC. We are systematically testing TSC1 and TSC2 gene therapy effectiveness and safety in animal models of TSC before we can begin human studies. As data is generated, we will keep the community informed of progress.
Help the TSC Alliance accelerate TSC research by donating to the 50 Forward Fund!