Mutations in the isocitrate dehydrogenase enzymes IDH1 and IDH2 have been associated with oncogenic transformation in several malignancies, including gliomas, acute myeloid leukemia (AML), cholangiocarcinoma and chondrosarcomas. Despite the approval of several mIDH1 inhibitors, suboptimal blood-brain barrier penetration and the development of acquired resistance limit their use.

Cantoni Therapeutics BV has announced the closing of additional financing to advance its lead asset into IND-enabling studies for obesity and its comorbidities. The company is developing small-molecule inhibitors of nicotinamide N-methyltransferase (NNMT), a promising target for cardiometabolic diseases. Cantoni has also won a Novo Nordisk ValidatioNN grant supporting translational research work on the mode of action of NNMT inhibition.

Rona Therapeutics Co. Ltd. has announced an IND filing with the Australian Human Research Ethics Committee (HREC) for RN-3161 for obesity. RN-3161, a novel siRNA developed through Rona’s proprietary GAIA platform, targets INHBE to reduce fat while preserving lean mass.

An ongoing concern for scientists is that there will be across-the-board funding cuts. This is already happening in mRNA research, where reductions affected coronavirus-related projects. During the pandemic, efforts focused on this pathogen, and once the health emergency was over, grants for antivirals were eliminated. However, these drugs could stem future outbreaks. Despite the cuts, recent research continues to demonstrate the potential of mRNA, not only for the development of antivirals, but also for obtaining more effective and longer-lasting vaccines.