Researchers in the U.K. have succeeded in reverse engineering the defective cryptic splicing that drives amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) to enable precisely targeted delivery of transgenes and therapeutic protein expression in diseased neurons. The technique is compatible with conventional adeno-associated viral vectors that are approved for gene therapy, and can readily be adapted for different transgenes. ALS, FTD and other neurogenerative diseases are underpinned by loss of function of the RNA-binding protein TDP-43 (transactive response DNA-binding protein 43), that normally functions as a key regulator of splicing, protecting the transcriptome from toxic cryptic exons.

Researchers from Stanford University have filed for protection of their use of mechanotransduction inhibitors in coatings for surgical sutures to reduce scarring.

An innovation that has kickstarted a revolution in the study and practice of health care is getting even more attention. Three scientists who pioneered the discovery and development of glucagon-like peptide-1 (GLP-1)-based obesity treatments have been named 2024 Lasker Award winners.

Axcelead Drug Discovery Partners (DDP) Inc. has entered into a strategic research and collaboration agreement with Eli Lilly & Co. for multiple drug discovery programs.

Nanovation Therapeutics Inc. has established a multiyear partnership with Novo Nordisk A/S to advance the development of novel genetic medicines targeting cardiometabolic and rare diseases. The partnership brings together Nanovation’s proprietary long-circulating lipid nanoparticle (lcLNP) technology for RNA delivery to cells outside of the liver, and Novo Nordisk’s expertise in cardiometabolic and rare disease R&D and clinical translation.

X-Chem Inc. has announced the achievement of a drug discovery milestone under its collaboration agreement with Astrazeneca plc.

Rakovina Therapeutics Inc. and Variational AI Inc. have entered into a research collaboration to identify and develop novel small-molecule therapies against DNA damage response (DDR) targets for the treatment of cancer.

C4 Therapeutics Inc. has successfully delivered a second development candidate to Biogen Inc. under the companies’ 2018 strategic collaboration.