Eye On the Cure Research News

Nov 18, 2020

AGTC Announces Results for Achromatopsia Gene Therapy Clinical Trials

The company will continue enrolling younger patients in higher dosing groups.

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Applied Genetic Technologies Corporation (AGTC), a biotechnology company developing gene therapies for rare diseases, reported that some patients in its Phase 1/2 clinical trials for achromatopsia (caused by mutations in CNGA3 and CNGB3) showed vision improvements in at least one measure of visual function, but no consistent, sustained improvements were observed. Some patients reported vision improvements through anecdotal statements and surveys.

Achromatopsia is an inherited retinal disease that causes extreme sensitivity to light as well as significant loss of visual acuity and color perception. The condition can be caused by mutations in one of six genes with mutations in CNGA3 and CNGB3 being the most common cause.

AGTC plans to continue enrollment of pediatric patients in the two highest dose groups of the ongoing Phase 1/2 achromatopsia gene therapy clinical trials and has amended the study protocol to allow enrollment of patients as young as four years of age and to include additional assessments such as functional MRI and improved color brightness tests. The company believes that longer treatment durations and/or treatment of younger patients may yield better results.

The company reported 12-month achromatopsia gene therapy clinical trial data in the original three dose groups (low, medium and high), as well as six- to nine-month data at two higher dose groups (higher and highest).

Also, on November 11, AGTC reported additional positive data from its Phase 1/2 gene therapy clinical trial for patients with X-linked retinitis pigmentosa (XLRP) that indicated durable improvements observed in visual sensitivity and visual acuity over a wide dose range with a favorable safety profile out to month 12 in two of the dose groups. The company is planning a Phase 2/3 clinical trial of its XLRP gene therapy in 2021.