AGTC Announces Topline Interim Six-Month Data from Phase 1/2 X-Linked Retinoschisis Clinical Study

Applied Genetic Technologies (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today reported topline interim six-month data from its Phase 1/2 clinical trial of rAAV2tYF-CB-hRS1, an investigational AAV-based gene therapy delivered via intravitreal injection for X-linked retinoschisis (XLRS) due to mutations in the RS1 gene.

As quoted in the press release:

AGTC also announced that the company will regain full rights to the XLRS and X-linked retinitis pigmentosa (XLRP) programs and the three other partnered discovery programs following Biogen’s termination of the collaboration agreement, which will be effective on March 8, 2019. The company will complete patient monitoring activities on the XLRS program according to the clinical protocol but will not further develop the product. AGTC plans to advance the XLRP program as previously disclosed, and will determine next steps for the three discovery programs over the next several months.

“The data from the XLRS trial support the general safety and tolerability of our gene delivery platform and further reinforce our confidence in our ongoing Phase 1/2 trials in achromatopsia (ACHM) and XLRP,” said Sue Washer, President and CEO of AGTC. “We are extremely grateful for the commitment of the patients, families, patient advocacy organizations and our clinical collaborators, which was crucial to completing enrollment of this Phase 1/2 trial. Through this study, we have deepened our understanding of the challenges that patients with inherited ophthalmic diseases face every day. It has also provided us with valuable information and insights in designing and analyzing clinical trials for inherited retinal diseases. We remain dedicated to leveraging our gene delivery platform to improve the care and outcomes for patients with ACHM, XLRP and other ophthalmic diseases.”

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