Today, AviadoBio announced the start of its Phase 1/2 clinical trial to evaluate AVB-101 as a one-time treatment for candidates who have the progranulin (GRN) genetic variant. AFTD applauds AviadoBio’s innovative approach to treating frontotemporal degeneration (FTD).

AVB-101 is an investigational gene therapy that will be delivered using a minimally invasive neurosurgical procedure on the part of the brain called the thalamus. This delivery method will reduce the dose required for effectiveness and limit potential systemic exposure.

The first two countries with testing sites are Poland and Spain, but the trial is expected to expand to multiple locations in Europe and in the U.S. Click here for more information about the trial.

“The field of FTD is lucky to have AviadoBio and other innovative companies testing potential treatments for FTD caused by the progranulin mutations,” said AFTD Senior Director of Scientific Initiatives Penny Dacks, PhD. “Each treatment approach is different, and each trial is a critical step towards slowing, stopping, and eventually preventing this horrible disease from devastating families.

“While the people with FTD-GRN mutations are actively sought for clinical trials today, what we learn from these trials will accelerate the development of treatments for all people with FTD,” Dr. Dacks added.

More information about progranulin-related clinical trials can be found here.

For those wishing to take action today, AFTD encourages you to enroll in the FTD Disorders Registry to be notified of clinical research studies for which you or a family member may be eligible.

In addition, if a loved one is currently diagnosed with FTD, consider genetic counseling. Many current trials for FTD seek people with specific genetic causes of FTD, particularly FTD-GRN. Genetic counseling can empower informed decision-making on whether genetic testing or genetic banking is a good option for your family.