AGTC Announces Publication of Positive Preclinical Data that Provides Foundational Scientific Evidence for its Ongoing Clinical Program in Patients with X-Linked Retinitis Pigmentosa (XLRP...
Applied Genetic Technologies Corporation (AGTC)
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Source: GlobeNewswire
- Administration of AGTC’s proprietary adeno-associated virus (AAV) vectors expressing variants of the RPGR gene corrected early markers of disease and provided evidence of a rescue effect in an animal model of XLRP, with a favorable safety profile - GAINESVILLE, Fla., and CAMBRIDGE, Mass., May 20, 2020 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced the publication of positive preclinical data that provide the foundation for the vector and starting dose used in its ongoing Phase 1/2 clinical trial in patients with XLRP due to mutations in the RPGR gene. The data, which demonstrate that the company’s proprietary AAV vector and engineered RPGR constructs were well tolerated and had beneficial effects on markers of disease in a canine model of XLRP, were published online in Human Gene Therapy.
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