Applied Genetic Technologies Corporation has completed enrollment in all dose groups for adult patients with achromatopsia in its phase 1/2 clinical programs, according to a press release.
The company is evaluating the safety and efficacy of rAAV2tYF-PR1.7-hCNGA3 in patients with achromatopsia caused by mutations in the CNGA3 or CNGB3 gene and continues to enroll pediatric patients at higher doses in both trials.
Interim 6-month data reported in January from the dose escalation cohorts of the ongoing clinical trials demonstrated signs of biologic activity as shown by positive changes in light discomfort testing and patient anecdotes describing real-world improvements in visual function, according to a press release.
“We plan to report interim data from all adult dose groups in the second half of 2020 and to use the data to inform decision-making regarding readiness to move the product candidates to pivotal trials. In addition to being on track for our end of phase 2 submission for our X-linked retinitis pigmentosa clinical study, we expect to have multiple achromatopsia data readouts in 2020 that will build on the momentum we created in January,” Sue Washer, president and CEO of AGTC, said in the release.