Press Release

March 15, 2021
1 min read

FDA grants orphan drug designation for retinitis pigmentosa treatment


Press Release

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

The FDA has granted orphan drug designation for chemically induced photoreceptor-like cells to treat retinitis pigmentosa, according to a press release from CiRC Biosciences.

The technology “enables direct chemical transdifferentiation of fibroblasts into other cell types using a cocktail of small molecules in a chemical conversion process that takes less than 2 weeks,” the release said.

Sign outside FDA HQ in Washington, DC.
Source: Adobe Stock

A study conducted in an animal model of retinitis pigmentosa found that chemically induced photoreceptor-like cells led “to partial restoration of the pupil reflex and visual function,” the release said.

The cells are also in preclinical stages of development for geographic atrophy age-related macular degeneration and advanced retinitis pigmentosa, the release said.

“Utilizing CiRC Biosciences’ cell therapy to potentially restore vision in those living with vision loss caused by retinopathies and macular degeneration advances our mission to find solutions to some of the world’s greatest challenges,” Jeff Aronin, co-founder of CiRC Biosciences and chairman, founder and CEO of Paragon Biosciences, said in the release.