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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

uniQure’s Huntington gene therapy receives FDA’s fast track designation

The FDA has granted uniQure a fast track designation for its gene therapy candidate, AMT-130 designed to target the root cause of Huntington’s disease.

Huntington disease is a rare and fatal neurodegenerative genetic disorder caused by the expansion of CAG trinucleotide in exon 1 of a gene that codes for huntingtin protein. The disease affects motor and cognitive function, leading to total physical and mental deterioration.

There are no approved therapies to treat the disease, delay the onset or slow its progression. This, and the rarity of the disease coupled with the innovation of the therapy have prompted the FDA to grant fast track designation to AMT-130. The product also received FDA’s orphan drug designation and EMA’s Orphan Medicinal Product Designation.

The mechanism by which AMT-130 targets Huntington’s disease is by silencing the huntingtin gene using an artificial microRNA which is delivered directly to the brain using an AAV5 vector. Another striking feature of the therapy is that it targets the highly toxic exon 1 protein fragment of huntingtin protein that is even more toxic than the mutant huntingtin.

FDA has now completed the review of the IND application of AMT-130 and the company intends to dose the first patient in the Phase 1/2 trial in the second half of 2019. If all goes according to the plan, it will be the first one-time administered AAV gene therapy to enter clinical trial for treating Huntington’s disease.

Source: Gene Therapy Fast-Tracked for Huntington’s Disease Treatment; Press Release

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