AGTC Completes Enrollment of Third Group in the Dose Escalation Portion of the Achromatopsia CNGA3 Phase 1/2 Clinical Study

UF startup and UF Innovate│Sid Martin Biotech graduate Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced that it has completed enrollment of the third group in the dose-escalation portion of its Phase 1/2 clinical trial evaluating the safety and efficacy of subretinal injection of (rAAV2tYF-PR1.7-hCNGA3) for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene.

“This achievement is another demonstration of our continued progress in the clinical development of our three product candidates for the treatment of X-linked retinitis pigmentosa and achromatopsia due to mutations in the CNGA3 or CNGB3 gene,” said Sue Washer, president and CEO of AGTC. “To date, we have dosed 68 patients across our orphan ophthalmology programs. This reflects the strong enthusiasm and support among patients and physicians for novel approaches to treating diseases that have a tremendous unmet medical need.”

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