FDA grants orphan drug designation for retinitis pigmentosa treatment
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.
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.