It will commercialise two drug candidates based on research at Colorado University.

Ocugen, a Colorado-based startup, has signed a licensing agreement with Colorado University to continue development of two drug candidates for the treatment of rare eye diseases. One of the two candidates, OCU100, has already received orphan drug status from the US Food and Drug Administration (FDA).

OCU100 will be used to treat retinitis pigmentosa. The disease, for which there is currently no treatment, is an inherited, degenerative eye disease that causes severe vision impairment and often blindness. Although the disease progress is not consistent, and some people suffer from the onset earlier in life than others, most people with the disease are legally blind by age 40. About 100,000 people in the US are affected.

Orphan drug status is granted by the FDA’s Office of Orphan Products Development for novel drugs that treat a rare disease or condition affecting fewer than 200,000 patients in the US. These rare conditions are known as orphan diseases. The designation qualifies the sponsor of the drug for various development incentives under the Orphan Drug Act, such as a seven-year period of US marketing exclusivity, tax credits for qualified clinical testing, waiver of prescription drug user fee for marketing application, and ability to apply for grants.

The other drug candidate licensed to Ocugen is a treatment for wet age-related macular degeneration (AMD). It is an advanced form of AMD, which causes vision loss due to abnormal blood vessel growth ultimately leading to blood and protein leakage below the macula, near the centre of the retina. Bleeding, leaking, and scarring from these blood vessels eventually cause irreversible damage to the photoreceptors and rapid vision loss if left untreated.

Uday Kompella, founder and board member, said: “OCU100 has shown potential as a promising therapeutic agent for treating retinitis pigmentosa by reducing protein aggregation and associated cellular stresses, which are known to contribute to this condition. With impressive preclinical data, we look forward to progressing with a phase 1 study for safety and tolerability in patients sometime in 2015.”