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Applied Genetic reaches enrollment milestones in two clinical trials.

Applied Genetic announced that it has achieved enrollment milestones in two of its Phase 1/2 clinical trials. The company completed enrollment of the dose escalation portion of the achromatopsia, or ACHM, CNGB3 trial, which is evaluating the safety and efficacy of its product candidate, rAAV2tYF-PR1.7-hCNGB3, for the treatment of ACHM caused by mutations in the CNGB3 gene. The company also completed enrollment in the expansion group, which includes pediatric patients, of its product candidate, rAAV2tYF-GRK1-RPGR, for the treatment of X-linked retinitis pigmentosa, or XLRP, caused by mutations in the RPGR gene. Both investigational therapies utilize the company's proprietary AAV delivery technology and are administered by subretinal injection. The company is enrolling patients in two parallel Phase 1/2 clinical trials of its product candidates for ACHM caused by mutations in the two most common ACHM genes, CNGB3 and CNGA3. To date, AGTC has enrolled 12 patients in the ACHM CNGB3 trial and six patients in the ACHM CNGA3 trial. AGTC plans to provide topline interim six-month data for the dose escalation portion of the CNGB3 study in the second half of the year. To date, AGTC has completed enrollment of 10 and six patients in the dose escalation and expansion portions of the XLRP trial, respectively. The company plans to provide topline interim six-month data for the dose escalation portion of the trial in Q3 and the expansion portion in Q4.

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Publication:The Fly
Date:Apr 8, 2019
Words:233
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