Antisense Oligonucleotide

Antisense RNA therapy shows early promise in rare ALS case, plus US sanctions linked to Chinese patent surge

An antisense oligonucleotide therapy targeting the faulty CHCHD10 gene produced improved symptoms in the first ALS patient treated with it, who remained functional as a physician a year later. Separately, a study found that Chinese companies placed on the US Entity List appear to have increased their science patents and publications compared with non-sanctioned firms, though causation remains unproven.

nature.com · 2026-09-18

Personalized RNA drug shows early promise in rare ALS case

A man with a slow-progressing, genetically rare form of ALS became the first person to receive an antisense oligonucleotide therapy designed specifically to target his causal mutation. A year later, he reported improved symptoms and remains able to work as a physician, according to results published in Med.

nature.com · 2026-09-18