AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

November 6, 2023

• AVB-101 is an investigational gene therapy delivered as a one-time infusion directly to the brain, enabling distribution to brain areas affected by FTD while limiting systemic exposure

• The Phase 1/2 ASPIRE-FTD global clinical trial is currently open in Europe and will open in the U.S. in 2024

• FTD is a leading cause of dementia in people under the age of 65

London, UK; November 6, 2023 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AVB-101 to be studied in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene. In addition, the FDA has granted Fast Track designation to AVB-101 for the treatment of FTD-GRN to slow disease progression. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. Fast Track designation is intended to bring new medicines to patients earlier.

AVB-101 has been designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to areas of the brain affected by FTD. Both the U.S. FDA and the European Commission (EC) granted orphan designation to AVB-101 for the treatment of FTD in 2022. The company recently announced enrollment is open in European countries for ASPIRE-FTD, an open-label, multi-center dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN.

“The IND clearance and Fast Track designation of AVB-101 are important milestones for the FTD community and for our company. Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations. Receiving Fast Track designation underscores the significant need for treatment options for these patients, and we are eager to soon open U.S. clinical trial sites and offer an innovative option for eligible patients with FTD-GRN,” said Lisa Deschamps, Chief Executive Officer.

“We are using adeno-associated virus (AAV) technology and taking a novel approach to deliver a functional copy of the GRN gene directly to the brain via bilateral, MRI-guided intrathalamic infusion with the aim to restore normal levels of the progranulin protein,” said David Cooper, M.D., Chief Medical Officer. “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.”

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.¹,² People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³ FTD is a leading cause of dementia in people under the age of 65⁴ with an estimated prevalence at any one time of up to 4.6 cases per 1,000 of people.⁵ Genetic FTD cases account for about one-third of cases and are most frequently associated with autosomal dominant mutations in three genes, including the progranulin gene.⁶

1. Onyike CU. Neuroepidemiology. 2011;37:166–167;
2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310;
3. Pressman P and Miller BL. Biol Psychiatry. 2014;75(7):574–581;
4. Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216;
5. Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl 1):S96–S109;
6. Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48.

All News & Insights