(CercleFinance.com) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced this weekend the main results of the phase 1/2 study evaluating the experimental gene therapy botaretigene sparoparvovec (formerly AAV-RPGR) in patients with the disease hereditary retinal XLRP, a rare disease that causes progressive loss of vision.
Treatment with botaretigene sparoparvovec was found to have an acceptable safety profile, and efficacy ratings were demonstrated in this proof-of-concept study to improve improvements in retinal sensitivity, visual function, and vision functional.
These results and additional updates were presented in oral presentations at Retina Subspecialty Day at the American Academy of Ophthalmology (AAO) Annual Meeting.
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