Applied Genetic Technologies (AGTC) and Bionic Sight are teaming up to develop a new form of optogenetic therapy that can restore vision in patients who have visual deficiencies or are blind as a result of retinal disease. The technology will combine Bionic Sight’s neuroprosthetic device and retinal coding algorithm with ACTC’s ophthalmology gene therapy expertise to stimulate patients’ remaining healthy retinal cells and restore normal neuronal signaling.
The Bionic Sight device combines a camera with proprietary software that can transform incoming images into the retinal code that the brain uses to convert electrical signals from photoreceptor cells, through the retina to the brain. The partnership between Bionic Sight and AGTC aims to use the device to activate an optogenetic protein delivered using AGTC adeno-associated virus (AAV) gene therapy and trigger the target cells to fire and recreate images that the brain can detect.
“AGTC is excited to combine our expertise in gene therapy and ophthalmology with Bionic Sight's transformative device that incorporates the retina's neural code to improve the lives of patients with retinal disorders,” said Sue Washer, president and CEO of AGTC. “Bionic Sight has demonstrated an ability to mimic normal image formation in preclinical studies, and we believe there is a potential to achieve an outcome for patients that greatly exceeds what is currently possible with prosthetic or gene therapy approaches in late-stage degenerative retinal diseases.”
Bionic Sight is an early-stage medical device company focused on the development of prosthetic treatments for blindness causd by retinal degenerative disease. The firm’s founder, Sheila Nirenberg, Ph.D., professor in the department of physiology and biophysics at Weill Medical College of Cornell University, added, “We're very enthusiastic about this opportunity to collaborate with AGTC, and recognize the potential of their proprietary gene delivery technology to support our clinical development program. AGTC's AAV-based delivery platform has been validated in multiple preclinical and clinical studies, and we believe our combined technologies and capabilities have the potential to contribute significantly to patients affected by severe vision impairment.”
AGTC is exploiting its AAV gene therapy platform to develop a pipeline of six early clinical- and preclinical-stage ophthalmology gene therapies against X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia (ACHM), wet age-related macular degeneration, and blue cone monochromacy. Two nonophthalmology programs are in development for the potential treatment of alpha-1 antitrypsin deficiency and adrenoleukodystrophy. The XLRS program and one of two achromatopsia candidates are in early clinical development. A second achromatopsia candidate is anticipated to start in clinical development during 2017. AGTC is developing the XLRS and XLRP programs in collaboration with Biogen, under a broad, $124 million up-front collaboration and license agreement signed in July 2015.