On 15 April, the UK-based biotechnology company AviadoBio announced the initiation of its Phase I/II ASPIRE-FTD trial evaluating AVB-101 in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain. The open-label and multi-centre trial aims to evaluate the safety and efficacy of AVB-101, which is administered directly to the part of the brain called the thalamus. AVB-101 was administered to the first participant in the ASPIRE-FTD trial at the Interventional Neurotherapy Center at Mazowiecki Szpital Bródnowski Hospital in Warsaw, Poland.
Clinical trial sites are currently open in Poland, Spain and the Netherlands. The company plans to expand the trial to additional countries, including the US. In November last year, AVB-101 received fast-track designation from the US Food and Drug Administration (FDA). 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. AVB-101 has also received orphan drug designation by both the FDA and European Commission.
https://aviadobio.com/first-patient-treated-in-aspire-ftd-clinical-trial/
