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Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine

Demaris Mills (Forbes)

January 12, 2024


The 2023 Oligonucleotide Therapeutics Society meeting drew 800 attendees to Barcelona, a clear sign that nucleic acid-based drugs are becoming mainstream, especially the oldest technology in this class, antisense oligonucleotides (ASOs). Buzz at the conference centered around the potential of ASO technologies to enable personalized treatments for ultra-rare diseases, many of which are untreatable using traditional small molecule drugs. With a new era of nucleic acid-based drugs and genetic medicines having arrived, many of these diseases could now become treatable.


ASOs work by targeting RNA to alter gene expression, which can modify the expression level or structure of disease-causing proteins in ways that small-molecule drugs can't. Ten ASO drugs have been approved by the FDA or European Medicines Agency (EMA) since this therapeutic class emerged in the late 1990s, including four that treat Duchenne muscular dystrophy (eteplirsen, golodirsen, vitolarsen and casimersen) and one that treats the neuromuscular disorder spinal muscular atrophy, or SMA (nusinersen).

Many ASO researchers believe this technology holds great promise for the development of “n-of-1” treatments—drugs that are tailored to individual patients based on genetic mutations that are unique to them. One example is milasen, which was developed to treat a variant of Batten's disease. To realize more successes, biotechnology and pharmaceutical companies, academic medical centers, private foundations and regulatory agencies must first solve several challenges hindering rapid ASO advances. Here are four issues to consider.





Mills, D. (2024, January 15). Council post: Four keys to advancing antisense oligonucleotides for personalized medicine. Forbes. https://www.forbes.com/sites/forbestechcouncil/2024/01/12/four-keys-to-advancing-antisense-oligonucleotides-for-personalized-medicine/?sh=322c2ce864db

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