Bionic Sight Doses First Patient in a Phase 1/2 Clinical Trial of a New Investigational Treatment for Blindness 

Bionic Sight, LLC recently announced it has dosed the first patient in the company’s Phase 1/2 clinical trial of BS01, an optogenetic gene therapy for patients with Retinitis Pigmentosa. This is an important step toward bringing BS01 forward as a treatment to enable patients with retinal degenerative diseases to detect light, motion, and shapes, with the ultimate goal of restoring eyesight.

Optogenetic proteins act essentially as converters; they receive light and convert it into neural signals. Bionic Sight’s therapy delivers the optogenetic proteins to the optic nerve, which sends the signals to the brain.

What makes Bionic Sight’s treatment different from other optogenetic therapies is that it combines it with a device that drives the optic nerve to send out the same signals as a normal optic nerve would send. “A key issue for producing meaningful vision is to send signals to the brain that the brain can understand. When the optic nerve communicates with the brain, it does it in a code. The code is in the form of patterns of neural pulses that tell the brain what you’re looking at – faces, objects, a clear path ahead of you,” explains Dr. Sheila Nirenberg, Professor of Physiology and Biophysics at the Weill Medical College of Cornell University and a recipient of a MacArthur “Genius” Award for deciphering the retina’s neural code. “If you can deliver signals in the language the brain understands, then meaningful perceptions become possible.”

“This is an exciting clinical milestone for one of the first optogenetic treatments for a retinal degenerative disease, and we are excited to be in a collaborative partnership with Bionic Sight,” said Sue Washer, CEO of Applied Genetic Technologies Corporation, a clinical-stage biotechnology company that develops novel gene therapies to treat debilitating diseases, primarily in the field of ophthalmology. “We are committed to advancing novel gene therapies for inherited retinal disorders, and Bionic Sight’s unprecedented approach has the potential to transform the lives of patients.”

The company’s Phase 1/2 trial is an open-label, dose-escalation study in patients diagnosed with advanced-stage RP. Trial participants will be enrolled sequentially in four groups. Individuals in Groups 1, 2 and 3 will receive a low, middle and high dose of BS01, respectively. Patients in Group 4 will receive the maximum tolerated dose as determined by the first three groups. The primary focus of the study will be to assess the safety of BS01 through analysis of ocular or systemic treatment-emergent adverse events. Efficacy will be measured by assessing the detection of light, motion, and shape/object recognition.

The first patient was dosed in early March, 2020, but application of the therapy to subsequent patients was postponed due to the COVID-19 pandemic. Bionic Sight expects to resume enrollment in the second half of 2020. The trial is listed on ClinicalTrials.gov with the identifier: NCT04278131.

BIONIC SIGHT is a technology company that develops optogenetic gene therapy vectors and devices to treat retinal degenerative diseases. The company leverages the research of Sheila Nirenberg, a professor at the Weill Medical College of Cornell University, whose work focuses on neuroscience and its applications to brain/machine interfaces and computer vision. Dr. Nirenberg is the founder of Bionic Sight. Her work on deciphering the retina’s neural code has been described in TED talks, an NBC documentary, a Bloomberg documentary, the Discovery Channel, Scientific American, as well as peer-reviewed journals and patents. For more information, visit https://www.bionicsightllc.com.

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic, and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. Initially focusing on ophthalmology, their goal is to preserve or, hopefully, be able to improve vision in some cases. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Their preclinical programs build on their industry leading AAV manufacturing technology and expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in ophthalmology, otology and CNS disorders.