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Retinitis pigmentosa is an inherited eye disease which affects the retina, and impacts around 1.5 million people globally. In Vision Executives’ article on gene therapy, which was published in the The Ophthalmologist Magazine, we explored how some companies were aiming to use gene therapy to treat retinitis pigmentosa. Applied Genetic Technologies Corporation (AGTC) are a recent addition to this selection of companies who are aiming to treat the disease with gene therapy.

AGTC have released their 3-month interim data from their phase 2 Skyline trail of AGTC-501, an AAV viral vector-based gene therapy which targets the RPGR gene in patients with X-linked retinitis pigmentosa (XLRP). The results showed improvements in visual sensitivity in multiple patients after 3 months, the trials primary efficacy endpoint.

As with their phase 1/2 results, AGTC-501 was well tolerated with no clinically relevant safety concerns. ATGC believe this favourable safety profile is due to a strong focus on product design, extensive preclinical testing and enhanced inclusion and exclusion criteria.

“We are incredibly excited by the compelling interim results seen in the Skyline trial, including strong safety data and robust improvements in visual sensitivity with a clear difference between the two dose groups. These results add to the growing body of evidence supporting the best-in-class potential of AGTC-501 for the treatment of XLRP.” Sue Washer, President and CEO of AGTC.
AGTC-501 is an exciting development for the treatment of XLRP, a disease which currently has no FDA approved treatments.