First Patient Receives AVB-101 Gene Therapy in ASPIRE-FTD Trial for Frontotemporal Dementia Linked to GRN Gene Mutations

April 15, 2024 —An innovative gene therapy named AVB-101 is currently under investigation to treat frontotemporal dementia (FTD) caused by progranulin (GRN) gene mutations. This therapy aims to deliver a functional GRN gene to the brain, potentially halting disease progression. The ASPIRE-FTD trial, which marks a significant step for adult neurodegenerative diseases, has begun enrolling patients in Europe and the United States. The first patient has been treated in the Phase 1/2 ASPIRE-FTD trial, which is evaluating the safety and preliminary efficacy of AVB-101. AviadoBio, the company behind the therapy, is focused on developing transformative medicines for neurodegenerative disorders. The treatment is a potential one-time therapy that could stop the progression of FTD by restoring progranulin levels in the brain's affected areas. 

FTD is a severe form of early-onset dementia that often results in death within a decade of diagnosis. It is characterized by personality changes, behavioral issues, language loss, and reduced mobility. The condition is a leading cause of dementia in individuals under 65 and is frequently misdiagnosed. AVB-101 is delivered through a minimally invasive procedure directly to the thalamus, a brain region with extensive connections to areas critical for FTD symptoms. 

The Interventional Neurotherapy Center at Mazowiecki Szpital Bródnowski Hospital in Poland is the first European center to perform MRI-guided infusions of gene therapies, including the intrathalamic administration of AVB-101 in the ASPIRE-FTD trial. The therapy has received Fast Track designation from the U.S. FDA, which expedites the review process for drugs addressing serious conditions with unmet medical needs. 

The ASPIRE-FTD study is an open-label, multi-center, Phase 1/2 dose-escalation study. More details about the study can be found on the ClinicalTrials.gov website. AVB-101 is an investigational gene therapy containing a non-mutated version of the GRN gene, designed to restore brain progranulin levels and potentially slow or stop the progression of FTD-GRN. FTD is a leading cause of dementia in younger individuals, with a significant impact on work, family, and finances. Genetic FTD cases, which include those with GRN mutations, account for about one-third of all cases. AviadoBio, founded on research from King's College London and the UK Dementia Research Institute, is dedicated to translating scientific breakthroughs into life-changing medicines for FTD and ALS patients. The company's leadership team has extensive experience in gene therapy development, delivery, and commercialization.

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