AviadoBio Announces FDA Clearance of IND and Fast Track Designation for Treatment of Frontotemporal Dementia with GRN Mutations
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AviadoBio, a gene therapy company committed to developing and delivering innovative medicines for neurodegenerative disorders, has announced that the FDA has approved the Investigational New Drug (IND) application for AVB-101. This drug will be studied in patients with frontotemporal dementia (FTD) who have mutations in the progranulin (GRN) gene.
FTD is a leading cause of dementia in people under the age of 65 and typically leads to death within seven to thirteen years of symptoms onset and three to ten years from diagnosis.
The experimental gene therapy AVB-101 is intended to slow disease progression. It contains a non-mutated version of the GRN gene designed to restore levels of progranulin in the brain. AVB-101 will be delivered as a ‘one-time dose’ directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.
“Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations,” said Lisa Deschamps, CEO of AviadoBio. “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.”
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The U.S. FDA and the European Commission (EC) have both granted orphan designation to AVB-101 for the treatment of FTD in 2022. The company has recently announced that enrollment is open in European countries for ASPIRE-FTD. It is an open-label, multi-center, dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN.
“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.
FTD affects a person’s personality, behavior, language, apathy, and mobility. It is a common cause of dementia in people under 65, with an estimated prevalence of up to 4.6 cases per 1,000 people at any given time. About one-third of FTD cases are genetic and are often associated with autosomal dominant mutations in three genes, including the progranulin gene.
Source: AviadoBio Press Release, received via Email
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