AGTC updates plan for retinitis pigmentosa gene therapy trials
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Applied Genetic Technologies Corporation has received written feedback from the FDA about its potential X-linked retinitis pigmentosa treatment and updated its development plan accordingly, according to a press release.
The feedback focused mostly on the design and execution of a registration trial and future submissions. AGTC is conducting clinical trials of adeno-associated virus-based gene therapies for rare diseases, including X-linked retinitis pigmentosa (XLRP).
“We are pleased with the productive feedback from the FDA and are modifying our development program based on their recommendations to advance our XLRP gene therapy candidate,” Sue Washer, president and CEO, AGTC, said in the release.
The plan features an expansion of the current phase 1/2 trial in which about 20 patients will be treated in two masked dosing arms to collect additional data, including a mobility test. Dosing is expected to begin in the fourth quarter.
The revised phase 2/3 trial design will likely include two masked active arms and a control group, with visual sensitivity serving as the primary endpoint. Supplemental endpoints will include a mobility test. This trial is expected to begin in the first quarter of 2021.
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