Interim Six-Month Data of RPGR Gene Therapy Shows Significant Vision Improvement in Patients Living with X-Linked Retinitis Pigmentosa

Click to Access Audio Press ReleaseRARITAN, NJ, July 17, 2020 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today six-month data from the ongoing Phase 1/2 trial (NCT03252847) of its investigational gene therapy for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP). The interim data showed that low and intermediate doses of the investigational adeno-associated virus retinitis pigmentosa GTPase regulator (AAV-RPGR) were generally well-tolerated and indicated significant improvement in vision. Initial data on the novel AAV-RPGR asset, jointly developed with MeiraGTx Holdings plc, will be available today as a late-breaker, pre-recorded oral presentation at the American Society of Retina Specialists (ASRS) 2020 Virtual Annual Meeting.In patients with XLRP, the photoreceptors in the eye that are responsible for converting light into signals that are sent to the brain function poorly, leading to degeneration of the retina and legal blindness in adulthood. The companies’ AAV-RPGR gene therapy is being investigated to treat the most common and severe forms of XLRP caused by mutations in the RPGR gene by preserving and improving vision and slowing retinal degeneration. Currently, there are no approved treatments for this condition. “There is an urgent need to deliver a transformational therapy for people living with XLRP who experience progressive visual loss from childhood with eventual blindness in early adulthood,” s...
Source: Johnson and Johnson - Category: Pharmaceuticals Tags: Innovation Source Type: news