Clostridioides difficile has been traditionally isolated from healthcare facilities' inpatients, but it is increasingly being identified in people who have not recently been hospitalized and is more and more found in community settings. Investigators from Perelman School of Medicine at University of Pennsylvania have developed an mRNA-LNP vaccine with promising results in preventing and controlling C. difficile infection.

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.