https://www.ophthalmologytimes.com
The early results of the phase 1/2a PIONEER gene therapy study showed that the combination of a new treatment, gene therapy and a medical device designed to treat end-stage non-syndromic retinitis pigmentosa (RP), was well tolerated. RP, an inherited disease with more than 100 known mutations, is characterized by progressive and irreversible photoreceptor degeneration that can result in blindness when patients are approximately aged 40 to 45 years. The novel approach, described above, aims to restore light sensitivity to the diseased retina.
