At the recent American Society of Gene and Cell Therapy (ASGCT) meeting, Wave Life Sciences Ltd. presented siRNAs designed to suppress expression of the liver gene inhibin subunit β E (INHBE). Human genetic data show that heterozygous INHBE loss-of-function carriers exhibit a healthy metabolic profile.

Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with Reborna Biosciences Inc. to generate RNA-targeting novel small molecules in the field of the central nervous system.

Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with Reborna Biosciences Inc. to generate RNA-targeting novel small molecules in the field of the central nervous system.

Researchers from Suzhou Siran Biotech Co. Ltd. presented the discovery and preclinical characterization of SA-1211, an N-acetylgalactosamine (GalNAc)-conjugated siRNA dimer targeting both hepatitis B virus (HBV) and PD-L1 gene expression, being developed as a potential new therapeutic candidate for the treatment of chronic hepatitis B (CHB).

Switch Therapeutics Inc. has announced its first development candidate, a liver-sparing APOE (apolipoprotein E) RNAi therapy for treatment of Alzheimer’s disease in APOE4 carriers. Switch’s conditionally activated siRNA (CASi)-APOE program is designed to knock down APOE in the CNS without affecting APOE in the liver, where it plays a vital role in systemic lipid homeostasis.

Clostridioides difficile is 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 developed an mRNA-LNP vaccine with promising results in preventing and controlling C. difficile infection.

Clostridioides difficile has been traditionally isolated from health care 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.

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.”