Current treatments for Graves’ hyperthyroidism (GH) and Graves’ orbitopathy (GO) are ineffective. Researchers assessed the preclinical in vitro efficacy of SYD-5115 (Byondis BV), a novel low-molecular-weight thyrotropin receptor (TSH-R) antagonist. The compound comprises an N-acetylated dihydropyrrole pyrimidine core with various substituents, including a difluorinated-spirocyclohexyl ring and a chiral carbamate group on the pyrimidine ring.

With CRISPR-Cas9 technology making its way toward clinical practice, laboratories are studying different gene-editing techniques, from base editors to prime editors, to correct mutations associated with various pathologies. Researchers at Tessera Therapeutics Inc. have been inspired by retrotransposons to develop a tool for editing DNA using RNA and reverse diseases such as phenylketonuria (PKU) or sickle cell disease (SCD).

Incretin mimetics have revolutionized the treatment of obesity and type 2 diabetes. These therapies have shown remarkable efficacy in promoting weight loss and improving glucose metabolism and cardiometabolic health. However, a significant drawback of these therapies is the concurrent loss of lean body mass (LBM), which can account for a substantial overall weight reduction (15% to 40%). The reduction in LBM affects resting metabolic rate, often leading to a weight loss plateau and other negative outcomes.