Keyword: Retinitis Pigmentosa
Applied Genetic Technologies’ $1 billion-plus gene therapy pact with Biogen has been torn up after their lead candidate failed a phase 1/2 trial.
Scientists have demonstrated how mutations in cell proteins known as “splicing factors” can cause the eye disease retinitis pigmentosa.
Recent biotech discoveries include a sensory protein at the heart of hearing and new insights into how CAR-T design affects cancer treatments.
After reprogramming a type of retinal cells, Mount Sinai scientists helped blind mice regain some visual function.
The gene-editing approach called CRISPR-Cas9 has been grabbing headlines for its potential to address diseases ranging from cancer to sickle cell disease—but scientists at the Salk Institute may have found a way to improve upon the technology. And they’ve shown it could hold promise for restoring some sight in adults who are blind.
Allergan has bought gene therapy startup RetroSense Therapeutics to gain an early clinical candidate to treat the rare vision disorder retinitis pigmentosa (RP).