progerinin + lonafarnib

**Progerinin + lonafarnib** is an investigational combination therapy designed primarily for the treatment of Hutchinson-Gilford progeria syndrome (HGPS), an ultra-rare premature aging disorder. **Lonafarnib** is a small molecule farnesyl transferase inhibitor currently FDA-approved for progeria, targeting the farnesylation of the aberrant lamin A protein *progerin* and thus preventing its pathological accumulation at the nuclear envelope. **Progerinin** is a newer small molecule under investigation that disrupts the interaction between progerin and lamin A, helping to further ameliorate cellular and nuclear abnormalities associated with the disease. The rationale for combining these agents is to target different steps in the progerin pathogenic pathway: lonafarnib blocks its post-translational modification, while progerinin disrupts deleterious protein interactions, with the aim of synergistically improving outcomes in HGPS[1][3][5].

Other names

FTI (for lonafarnib)progerinin (also sometimes called C75 or C3743 in some literature, but best-known as progerinin)