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RPGR Gene Therapy Shows Statistically Significant & Continued Vision Improvement

Late-Breaking 12-Month Data of Investigational RPGR Gene Therapy Shows Statistically Significant and Continued Vision Improvement in Patients with X-Linked Retinitis Pigmentosa


Nov 13, 2020, 17:55 ET

One-year findings support advancement into Phase 3 and include significant functional vision improvements observed in the time taken to walk through a vision-guided mobility maze at nine months

RARITAN, N.J., Nov. 13, 2020 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today new 12-month data from the ongoing Phase 1/2 trial (NCT03252847) of its investigational gene therapy for inherited retinal disease X-linked retinitis pigmentosa (XLRP). The data showed that low and intermediate doses were well-tolerated and continued to demonstrate statistically significant sustained or increased vision improvement across multiple metrics (mean sensitivity, volumetric and pointwise) and modalities (full-field static perimetry and microperimetry). Data on the novel adeno-associated virus retinitis pigmentosa GTPase regulator (AAV-RPGR), jointly developed with MeiraGTx Holdings plc, were presented today as a late-breaking study at the American Academy of Ophthalmology (AAO) 2020 Virtual Annual Meeting.

In patients with XLRP, the photoreceptors 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 for 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 available treatments.

"Living with XLRP is extremely devastating for patients and their families, as there is no treatment available and they live each day knowing they will eventually go blind," said Michel Michaelides,1 B.Sc., M.B., B.S., M.D. (Res), FRCOphth, FACS, trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital, Professor of Ophthalmology, University College London. "The continuous upward trend in efficacy we've observed through one year with this gene therapy is extremely promising as a potential way to halt the progression toward blindness in these patients."


Media Contacts: Sarah Freeman Phone: +1 215-510-4758

Jennifer Silvent Phone: +1 973-479-9845

Investor Relations: Christopher DelOrefice Office: +1 732-524-2955

Jennifer McIntyre Office: +1 732-524-3922

SOURCE Janssen Pharmaceutical Companies of Johnson & Johnson


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