Sun Pharmaceutical Industries, an international specialty pharmaceutical company focused on chronic diseases, and Intrexon, a synthetic biology company, have formed a joint venture to develop controllable gene-based therapies for the treatment of ocular diseases that cause partial or total blindness. Initial targets are dry age-related macular degeneration (AMD), glaucoma and retinitis pigmentosa.

The joint venture will leverage Sun Pharma's global capabilities and experience in developing and manufacturing complex dosage forms and specialty pharmaceuticals for niche therapy areas. The companies will share in both the financing and the revenues.

The venture will have access to Intrexon's full suite of proprietary synthetic biology technologies, including the RheoSwitch Therapeutic System (RTS) platform. RTS is a clinically validated method for controlling the location, concentration and timing of protein expression.  RTS may address a long-standing limitation of current approaches by enabling patients to receive a targeted biologic therapy without having to endure a lifetime of injections.

The companies intend to further expand the pipeline of targeted ocular diseases to potentially include wet AMD, macular edema, non-infectious uveitis and diabetic retinopathy.

Samuel Broder, M.D., senior vice president of Intrexon's health sector and former director of the National Cancer Institute, said current treatments for major ocular disorders require frequent and often painful interventions that, at best, only slow the progression of the disease.

"Using our RTS platform, the goal is to engineer a gene-based ocular treatment in which the concentration, location and timing of protein expression can be controlled with small-molecule therapy," Broder said. "In combination with Sun Pharma, we hope to produce a therapy that is minimally invasive as well as maximally effective in treating these debilitating diseases."

Dilip Shanghvi, managing director of Sun Pharma, said, "We believe Intrexon's biotechnology capabilities will enable us to develop not one, but several highly-effective, novel treatments for multiple ocular disorders where there currently is an unmet need.”