What Is Ss 31?

Visao Geral

SS-31 (elamipretide) e um(a) sintetico(a), aromatic-cationic tetrapeptide pertencente a the Szeto-Schiller (SS) family of mitochondria-targeted peptides. Its sequence — D-Arg-Dmt-Lys-Phe-NH₂ — alternates cationic residues (D-Arg, Lys) with aromatic residues (Dmt, Phe), an architectural pattern que confere selective, high-affinity binding to cardiolipin (CL) no(a) inner membrana mitocondrial (IMM).[1][2]

Discovery: SS-31 was identificado(a) serendipitously by Dr. Hazel Szeto (Weill Cornell Medical College) and Dr. Peter Schiller (Montreal IRCM) during research into opioid agonista do receptors. It was derivado(a) de SS-02 ([Dmt¹]DALDA), a synthetic opioid peptide analog, but projetado(a) para completely eliminate opioid receptor activity enquanto retaining the aromatic-cationic motif necessario(a) para mitochondrial targeting.[1]

Key distinguishing features: (1) Concentrates 5,000-fold no(a) IMM via cardiolipin binding; (2) Uptake is membrane-potential-independent (diferentemente de MitoQ, que requer intact electrochemical gradient); (3) Has no effect on healthy mitochondria — melhora bioenergetics apenas in aged/dysfunctional mitochondria; (4) Small, water-soluble, crosses the blood-brain barrier.[2][4]

Regulatory milestone: On September 19, 2025, the FDA granted Accelerated Approval to Forzinity™ (elamipretide) for Barth syndrome — a rare genetic disorder caused by TAFAZZIN gene mutations levando a cardiolipin deficiency. This made SS-31 the first drug ever authorized pelo(a) FDA specifically for Barth syndrome, and one do(a) primeiro(a) mitochondrially-targeted drugs to receive aprovacao da FDA for qualquer indication.[3]

Comparado(a) a MOTS-c — another mitochondria-targeted composto de pesquisa — SS-31 acts upstream at o nivel of cardiolipin e o(a) electron transport chain itself, rather do que via nuclear gene regulacao.[1]