UMass Chan Medical School licensed the exclusive global rights to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of Stargardt disease and Leber congenital amaurosis type 10 (LCA10) to Iveric Bio, An Astellas Company.
While recent forays into gene therapy treatments for other forms of inherited retinal disease have been encouraging, the relatively large size of both the LCA10 and Stargardt genes have been a roadblock to using AAV vectors for treatment. In 2017, Hemant Khanna, PhD, then an associate professor of ophthalmology & visual sciences at UMass Chan, and now vice president of preclinical ocular research at Iveric Bio, developed a “minigene” approach to deliver a shorter version of the gene using clinically standard AAV viral vectors.
This minigene approach allows scientists to deliver an abridged version of the mutated gene to patients using AAV viral vectors as delivery vehicles while still generating clinical results regardless of where the specific mutation occurs in the gene.
“We are excited to be partnering on this minigene approach to treating inherited retinal disorders using standard AAV vectors,” said Parth Chakrabarti, MBA, executive vice chancellor for innovation and business development at UMass Chan. “As part of the Astellas family of companies, Iveric Bio is uniquely positioned to take this research to the next stage of development.”
Stargardt disease is a rare eye condition that affects the macula, the part of the retina that provides sharp and clear vision. In order for a person to inherit Stargardt disease, both parents must pass down a mutation in the ABCA4 gene. Symptoms usually develop in children, teenagers and young adults. Patients with Stargardt have problems with their central vision. Images may be blurry, distorted or have dark areas. However, peripheral vision is usually not affected, though some people may have trouble distinguishing colors and it may take longer than usual for vision to adjust when going between bright and dark areas.
Leber congenital amaurosis, meanwhile, is a degenerative disease, caused by a group of recessively inherited genetic mutations that lead to an inability to make a light-sensitive protein in the retina. Mutations in the CEP290 gene are the most common cause of Leber congenital amaurosis, responsible for up to 25 percent of cases.
Iveric Bio is focused on the discovery and development of treatments for retinal diseases with significant unmet medical needs. It is part of Astellas Pharma Inc., a pharmaceutical company contributing toward improving the health of people around the world through the provision of innovative and reliable pharmaceutical products.
The partnership was facilitated by BRIDGE Innovation and Business Development at UMass Chan Medical School.
