Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its authors Victor Ambros and Gary Ruvkun the 2024 Nobel Prize in Physiology or Medicine.
Research into the regulation of gene expression experienced a significant breakthrough with the discovery of microRNA, small RNA molecules that do not code for proteins but control their translation. This finding has earned its discoverers – Victor Ambros and Gary Ruvkun – the 2024 Nobel Prize in Physiology or Medicine “for the discovery of microRNA and its role in post-transcriptional gene regulation.”
Arrowhead Pharmaceuticals Inc. announced its plans to advance its RNAi drug candidates ARO-INHBE and ARO-ALK7 into clinical trials for the treatment of obesity and metabolic diseases.
Airna Corp. Inc., of Cambridge, Mass., announced it has closed an oversubscribed $60 million financing round, bringing its total series A funding to $90 million.
Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.
Triastek Inc. and Biontech SE have entered into a research collaboration and platform technology license agreement for the development of RNA therapeutics for oral delivery based on Triastek’s Melt Extrusion Deposition (MED) 3D printing technology.
Large-scale human genetic studies have revealed that loss-of-function INHBE variants are associated with a lower abdominal to gluteofemoral fat ratio, improved metabolic profile, and reduced fasting glucose levels.
Molecure SA has confirmed the in vitro activity of a molecule binding to a new mRNA target within its mRNA discovery platform. This represents the discovery of another class of molecules binding to a second mRNA biological target, which has been confirmed in in vitro assays.
A preclinical trial in mice and ferrets of an experimental mRNA vaccine against the H5 highly pathogenic avian influenza (HPAI) virus generated neutralizing antibodies and prevented severe illness and mortality of the animals.
Achilles Therapeutics plc has established a research collaboration with Arcturus Therapeutics Holdings Inc. to evaluate best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines targeting clonal neoantigens.
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