Clinical Trials
DELIVERING THE FUTURE OF GENE THERAPY
At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into potentially life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).
Our clinical development program is built on the research of our co-founders and world-leading scientists. We work with an extensive network of leading neurologists, neurosurgeons and disease experts to evaluate our investigational gene therapies.
INVESTIGATIONAL GENE THERAPY AVB-101
AVB-101 is an investigational, adeno-associated virus-based (AAV) gene supplementation therapy for FTD patients with disease-causing mutations in the GRN gene. Designed as a one-time therapy to potentially halt disease progression, it delivers a functional copy of the GRN gene to restore progranulin levels in the brain. It is delivered by an intrathalamic infusion for targeted biodistribution with the aim to limit the treatment to only the brain itself, where it is needed. AVB-101 has been granted orphan designation by the US FDA and European Commission.

A Phase 1/2 open-label clinical study to evaluate the safety and preliminary efficacy of AVB-101 in people with a genetic sub-type of frontotemporal dementia (FTD-GRN)
“Being relentless means always striving for the best implementation of our plans and ensuring we deliver as individuals, and as a company as whole. We know that patients have limited options and time. Even when the path may not be clear, we navigate a way forward by being bold and tenacious in our decision making.”
David Cooper, MD
Chief Medical Officer

David Cooper, MD
Chief Medical Officer
AviadoBio policy on expanded access to investigational therapies