Ocugen said that the US Food and Drug Administration (FDA) has accepted its investigational new drug application (IND) to launch a first-in-human clinical trial of the OCU400 (AAV-NR2E3) gene therapy candidate for the treatment of retinitis pigmentosa caused by genetic mutations found in NR2E3 and Rhodopsin.

The IND acceptance by the FDA allows the US-based biotech company to initiate a phase 1/2 trial for evaluating the modifier gene therapy candidate for the treatment of the inherited retinal degeneration.

Shankar Musunuri — CEO and Co-Founder of Ocugen said: “We are delighted to advance OCU400 into clinical trials, which exemplifies our goal of offering new options to people with genetic diseases where none currently exist.

“We’re collaborating with leading centers in eye care and have been vital partners to getting our trial launched and receive patients. With this final decision by the FDA, we are embarking on a new pathway of care through this innovative gene therapy.”

According to Ocugen, its modifier gene therapy platform has been designed to target nuclear hormone receptors (NHRs) that control various functions within the retina, providing it the potential to address several different gene mutations and, in turn, multiple retinal diseases with a single product.

OCU400 has four orphan drug disease designations given by the FDA for the treatment of four different gene mutation-associated retinal degenerative diseases.

The European Medicines Agency (EMA) granted broad orphan medicinal product designation to OCU400 for the treatment of retinitis pigmentosa (RP) as well as Leber congenital amaurosis (LCA).

  AAV-NR2E3, gene therapy, OCU400, Ocugen, retinal degeneration, retinitis pigmentosa, Shankar Musunuri, US FDA, USA