UF startup Applied Genetic Technologies Corporation, 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 achieved enrollment milestones in two of its Phase 1/2 clinical trials. The company completed enrollment of the dose escalation portion of the achromatopsia (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 (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.
“Continued success enrolling in all our clinical trials is critical to advancing new and innovative gene therapies for patients with inherited retinal diseases that lack approved treatment options and we are pleased with the progress we have made,” said Sue Washer, president and CEO of AGTC. “Achieving these important milestones is a testament to the dedication of the AGTC clinical teams and our clinical investigators, underscoring the support that our investigational therapies have among the patient communities we seek to serve.”Learn more about AGTC Announces Reaching Enrollment Milestones in Phase 1/2 Clinical Trials.