FDA grants fast track designation to AviadoBio’s dementia gene therapy

07 Nov 2023
www.pmlive.com
Fast TrackOrphan DrugGene TherapyClinical Study
The US Food and Drug Administration (FDA) has granted fast track designation to AviadoBio’s experiential dementia gene therapy and given clearance for the candidate to be studied in a subset of frontotemporal dementia (FTD) patients.
AVB-101Food and Drug Administration (FDA)ast track process, which is designed to improve the efficiency odementiat development and accelerate the review of treatments for serious conditions.frontotemporal dementia (FTD)
AVB-101form of early-onset dementiFDAis the leading cause of dementia in people aged under 65 years and typically leads to death within seven to 13 years of symptom onset.
FTDetic FTD cases account for about one-third of cases and ardementiarequently associated with autosomal dominant mutations in three genes, including the progranulin (GRN) gene.
The poteFTDal one-time therapy AVB-101 has been designed as to halt disease progression by delivering a functional copy of GRN to restore levels of prograprogranulin (GRN) the brain affected by FTD.
AviadoBio’s chief medical officAVB-101id Cooper, said: “Our preclinical programme shows robust bio-distribution to the braiGRNreas where it is needeprogranuline levels of progranulin, potentiallFTDlowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects.”
AviadoBiond the European Commission both granted orphan designation to the candidate as a treatment for FTD in 2022, and AviadoBio recently announced that enrolment to its phase 1/2 of the candidate in patients with FTD-GRN had begun iFTD-GRNe.
LisaFDAschamps, the company’s chief executive officer, said: “Sadly, there are currently no disease-modiFTDng therapies aAviadoBioor the treatment of people living with FTD with progranulin mutations.FTD-GRN
"Receiving fast track designation underscores the significant need for treatment options for these patients, and we are eager to soon open US clinical trial sites and ofFTD an inprogranulintion for eligible patients with FTD-GRN.”
The FDA’s decision on AVB-101 comes shortly after the Alzheimer's Society raised concerns that not enough people are being diagnosed with young-onset dementia in England.FTD-GRN
A reFDAt from NHS EnglAVB-101ealed that 34,412 people in England have been diagnosed with young-onset dementia, but the Alzheimer’s Society has said tdementianumber of people affected may be closer to 53,206, based on estimates shared with other dementia charities.
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