The archetypal UbiB protein COQ8 has human homologues COQ8A and COQ8B, both with well-established connection to human disease, with inactivating mutations in COQ8A resulting in autosomal recessive cerebellar ataxia. Researchers from the University of Wisconsin-Madison and affiliated organizations have now recently reported the discovery of small-molecule inhibitors of COQ8A.

A strong clinical association has been observed between epilepsy and the development of depression, however, it is difficult to study individual molecular and cellular mechanisms underlying these comorbidities in animal models. Researchers from Rutgers University have aimed to develop a cell-type-specific monogenic mouse model of epilepsy and depression comorbidities, which could serve as a tool for identifying disease mechanisms as well as for target and drug screening.

Pepgen Inc. has announced new preclinical data supporting the progression into clinical trials of PGN-EDODM1, its product candidate in development for the treatment of myotonic dystrophy type 1 (DM1).