For tailored, single-person antisense oligonucleotides, FDA offers guidance on nonclinical testing
Over the last several years, a new wave of hyper-personalized therapies have quickly made their way into people, just one at a time, and often tailored to a specific genetic variant.
Success stories with these expensive to make, so-called “n-of-1” therapies – from 6-year-old Mila Makovec with Batten disease, a rare, inherited neurological condition to 3-year-old Ipek Kuzu with ataxia telangiectasia, a rare inherited disorder that affects the nervous and other systems – have caused the FDA to take notice, particularly as both are antisense oligonucleotide (ASO) treatments.
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