Applied Genetic Technologies Corporation (AGTC)

CEO Mark Shearman, MD, PhD, describes Applied Genetic Technologies Corp., known as AGTC, as a clinical-stage biotechnology company focused on developing its proprietary gene vector technology for treatment of orphan monogenic diseases of the retina.

AGTC signed an R&D partnership with Biogen to oversee biological development and commercialization of its platform, which Dr. Shearman described at OIS@AAO as a “significant IP portfolio” with a “broad” pipeline to deliver commercial agents in two to three years.

More than 100 patients are involved in Phase I and II trials. The programs in or near clinical development are:

• XLRS program – An Investigational New Drug (IND) application for X-linked retinoschisis (XLRS) was filed in early 2015 and the agent is now in Phase I/II trials with 27 patients enrolled. The ascending dose study should delivery clinical data in first-quarter 2017, Dr. Shearman said. The primary study endpoint is to define the maximum tolerated dose.
• Two programs for treatment of achromatopsia – The company has two candidates, ACHMB-B3 and A3. AGTC expects to file the IND for the B3 agent in late 2015, with the IND for the A3 agent expected in mid-2016, Dr. Shearman said. One-year Phase I/II clinical data on B3 are expected in 2017.
• XLRP program – An X-linked retinitis pigmentosa (XLRP) therapy is now in a genetic dog model with results of the toxicology study due late in 2015, and a primate vector is in development. Dr. Shearman said AGTC expects to file an IND for this program in 2017.

AGTC has made strides in its manufacturing capability, Dr. Shearman said. “We have improved the process to increase the yield approximately 25- to 30-fold, improving its cost-effectiveness as well as the time to produce the material for both preclinical and clinical testing,” he said.

Presenter: