AviadoBio will soon open U.S. clinical trial sites for an experimental gene therapy that could halt the progression of FTD when caused by a variant in the GRN gene, the company announced on November 6.

The therapy, known as AVB-101, delivers a copy of the GRN gene directly to the brain via a one-time, minimally invasive surgical procedure.

When functioning properly, GRN instructs the body to produce an important protein called progranulin. If GRN is not functioning properly due to a variant, the production of progranulin is suppressed; low levels of progranulin in the brain have been linked to the development of FTD.

“Our preclinical program shows robust biodistribution to the brain areas where it is needed to restore levels of progranulin, potentially slowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects,” David Cooper, MD, AviadoBio’s Chief Medical Officer, said in a press release.

The U.S. Food and Drug Administration recently approved AviadoBio’s Investigational New Drug application for AVB-101, clearing the way for a clinical trial, known as ASPIRE-FTD, to begin in the U.S. According to the London-based gene-therapy company, ASPIRE-FTD sites in the U.S. will open enrollment in 2024. (ASPIRE-FTD sites have already opened enrollment in Spain and Poland.)

Visit ClinicalTrials.gov to learn more.

“AviadoBio’s commitment to developing, trialing, and bringing to market FTD-halting therapies brings hope to so many families affected by this disease,” said AFTD CEO Susan L-J Dickinson. “AFTD applauds the many innovative researchers around the world currently working on these critical interventions.”

On October 27, the U.S.-based biotechnology company Alector Therapeutics announced that it completed enrollment for INFRONT-3, a Phase 3 clinical trial testing the safety and efficacy of the drug latozinemab, which has been shown in studies to increases progranulin levels by inhibiting a receptor that naturally degrades the protein.