Loss-of-function variants in the ELP1 gene are the most prevalent predisposing genetic factors in childhood medulloblastoma, accounting for about 30% of sonic hedgehog medulloblastoma (SHH-3 subtype).

Pulmonary fibrosis is a potentially deadly lung disease characterized by progressive scarring and impaired lung function, with limited treatment options and a poor prognosis. Previous work found that targeting the activation, but not the production, of latent transforming growth factor 1 (TGF-β1) may offer therapeutic benefit in this condition.

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.

Monte Rosa Therapeutics Inc. has gained IND clearance from the FDA for MRT-8102, a NEK7-directed molecular glue degrader being developed to treat inflammatory conditions linked to NLRP3, IL-1β and IL-6 dysregulation.

Deepcure Inc. has nominated DC-15442, an oral selective STAT6 inhibitor, as its second development candidate. DC-15442 is designed to replicate the safety and efficacy of dupilumab, while offering an oral alternative to regular subcutaneous injections.

Researchers at the Massachusetts Institute of Technology and Recursion Pharmaceuticals Inc. have released an open-source AI model that can predict the binding strength of small molecules as well as structures of proteins and biomolecular complexes. The model, which is called Boltz-2 and was released by the research team on the developer platform Github on June 6, addresses a major bottleneck in drug discovery with its improved ability to predict binding strengths.