Devoted to developing genetic therapies through the use of vector technologies and dosing for patients with inherited retinal diseases, AGTC has partnered with Biogen and Bionic Sight to ensure there are “multiple shots on goal,” said Steven Potter, chief business officer. AGTC is currently working on four orphan products that are in the clinic and a fifth program in optogenetics that should start in 2018.
X-linked retinoschisis is (essentially) a disease where missing structural protein results in poor vision that cannot be corrected with spectacles. AGTC’s program is now past the dose-ranging studies and has moved into the expansion group; to date the product seems to be safe and well tolerated. Enrollment in Phase I/II should be complete by the end of Q1 2018, with initial readout six months later.
“There’s a common theme in the products we pick,” Potter noted. “There’s a significant population, we have robust animal models, and we have clear and common endpoints.” Other areas of exploration include achromatopsia and X-linked retinitis pigmentosa. AGTC hopes to file an IND for its optogenetics program early next year. The Bionic Sight external, noninvasive system is designed to bypass damaged tissue in the retina to provide direct stimulation to the output cells to send visual information to the brain.