N4-Acetylation of cytidine (ac4C) is an mRNA modification that enhances cellular mRNA stability and translation. Most eukaryotic organisms catalyze ac4C using a homologue of human N-acetyltransferase 10 (NAT10). Recent work has suggested the involvement of alterations in NAT10-mediated ac4C in several diseases, including autoimmune disorders, infections, inflammation and cancer.

Mindimmune Therapeutics Inc. has been awarded a grant by Rhode Island Life Science Hub to accelerate preclinical development work on MITI-101 for the treatment of mild to moderate Alzheimer’s disease. The award will accelerate development work needed to start first-in-human studies.

Casma Therapeutics Inc. has nominated its first development candidate, CSM-101, a first-in-class TRPML1 agonist. CSM-101 is being developed for the treatment of Gaucher’s disease patients with Parkinson’s disease, with the potential to expand into GBA-associated Parkinson’s disease and broader Parkinson’s disease populations.

Chronic pain is a constant challenge to around 20% of the global population, and treatments to mitigate such pain often cause unacceptable side effects because the receptors and signaling pathways involved in pain sensing also drive necessary processes in the heart, lungs and liver. Opioid analgesics can be effective against chronic pain, but they can lead to tolerance and addiction.