Topic
siRNA, antisense oligonucleotides, mRNA and self-amplifying RNA as non-integrating alternatives to DNA-based gene therapy.
Weizmann and Regensburg researchers report in Cell that heat-loving archaea carry natural RNA phosphorothioates, the backbone chemistry used in RNA drugs. Developers get a natural counterpart to study, and the paper's title also points to the enzymes that install the marks.
Reality
MIT chemical engineers built an automated process that lets developers set a lipid nanoparticle's size and shape before testing which one performs best. It replaces bench trial-and-error, though the study does not show a chosen size reaches a chosen organ.
Reality
ST Pharm signed three oligonucleotide contracts worth $182 million this year, each above $50 million, a size it did not reach once last year. Its earnings case now rests on commercial reorders, with the largest contract tied by industry estimates to a single Ionis drug, Tryngolza.
Reality
Purified bacterial mRNA aggregates in a tube, roughly as physical chemistry predicts. Whitehead researchers report that real E. coli sequences interact less than computer-made synonyms, and read that gap as selection for solubility.
Reality
Artisan Partners' David Samra is telling Novartis's chairman to rebuild the team that does its deals, after a week of three Phase III setbacks in which one of the drugs actually met its primary endpoint.
Reality
Nagoya University and FUJIFILM report longer-lasting expression from capped circular RNA in a new lipid nanoparticle. The mouse data are directional; the duration numbers are not public yet.
Reality
Both fatalities disclosed so far came out of China's investigator-initiated trial route, the lightest-touch pathway in the field. US lawmakers were pushing to copy parts of it in June.
Reality