Progerinin

Progerinin is a small molecule drug developed as an optimized inhibitor of the interaction between progerin and lamin A, targeting the underlying molecular pathology of Hutchinson-Gilford Progeria Syndrome (HGPS). HGPS is caused by a mutation in the LMNA gene that leads to production of progerin, a toxic form of lamin A protein. Progerinin disrupts the binding between progerin and lamin A/C, thereby reducing nuclear deformation and ameliorating premature senescence phenotypes characteristic of HGPS. In preclinical studies, oral administration of progerinin extended lifespan, improved body weight, reduced cardiovascular pathology, and alleviated other aging features in mouse models of HGPS. The drug has shown efficacy both *in vitro* (patient cells) and *in vivo* (mouse models), with favorable safety profiles observed in advanced preclinical toxicity testing. Clinical trials are ongoing to assess its safety and optimal dosing in patients with HGPS[1][4][5][7][8].

Other names

progerin-lamin A binding inhibitor