GAINESVILLE, FL. – AGTC, a privately-held, clinical stage biotechnology company, announced that the company has been awarded an additional $657,699 in funding under the Small Business Technology Transfer Program of the National Eye Institute (NEI), a division of the National Institutes of Health. This grant will supplement ongoing work under the $8.4 million grant AGTC was awarded by the NEI earlier this year and will support expansion of the company’s preclinical development work for AAV-CNGB3, a gene therapy treatment for achromatopsia. Funds will be used to conduct additional studies in an animal model of the disease using the species-specific version of the CNGB3 gene, which is expected to provide safety and efficacy results that better predict responses in human clinical trials.
“The preclinical data generated to date in our achromatopsia program is very encouraging, and we believe that the work supported by this new grant will provide important information that will help to optimize the design of human clinical trials for AAV-CNGB3.”
“Establishing animal models that accurately predict safety and efficacy in human trials is essential to the responsible and successful development of novel therapies,” said Sue Washer, President and CEO of AGTC. “The preclinical data generated to date in our achromatopsia program is very encouraging, and we believe that the work supported by this new grant will provide important information that will help to optimize the design of human clinical trials for AAV-CNGB3.”
Achromatopsia (ACHM) is a rare, inherited genetic condition that affects about 22,000 patients in the U.S. and Europe. Patients demonstrate impaired visual acuity at birth. ACHM is caused by genetic mutations in certain photoreceptor cells in patients’ eyes; when these cells do not function properly patients lose their fine visual acuity. Most people with achromatopsia are legally blind, lack color discrimination and experience extreme light sensitivity, resulting in daytime blindness. There is no treatment for achromatopsia, although deep red tinted glasses or contact lenses can reduce symptoms of light sensitivity.
The potential treatment being tested uses an engineered adeno-associated virus (AAV), a man-made virus that delivers healthy copies of the ACHM gene to the cells of the retina, restoring activity of the protein that is defective in ACHM patients. A single treatment is expected to stop the disease for at least several years, perhaps a lifetime. AAV is a naturally-occurring virus that has never been associated with any human disease, and AGTC’s AAV delivery system is successfully being used in clinical trials of Leber congenital amaurosis that have restored vision in more than 50 adults and children who were virtually blind. Previous research has shown promising signs of efficacy in dog and mouse models of ACHM.
AGTC will be working with academic researchers at the University of Pennsylvania and Michigan State University.