ISIS 388626

ISIS 388626 is a second-generation antisense oligonucleotide (ASO) designed to inhibit the expression of sodium-glucose cotransporter 2 (SGLT2) in the kidney. By targeting SGLT2 mRNA, it reduces renal glucose reabsorption and increases urinary glucose excretion (glucosuria), aiming to lower blood glucose levels in patients with type 2 diabetes mellitus. The drug consists of a shortmer ASO with two terminal 2'-methoxyethyl modifications for enhanced stability and potency. Preclinical studies demonstrated dose-dependent reductions in SGLT2 mRNA, increased glucosuria without hypoglycemia, and no significant adverse effects on kidney function at therapeutic doses. In early clinical trials, transient increases in serum creatinine and urinary markers of tubular injury were observed at higher doses but were reversible upon discontinuation[1][3][5][6].

Other names

GPAM ASOGPAT1 antisense oligonucleotideGPAT-1 antisense oligonucleotideGPAT 1 antisense oligonucleotidemitochondrial glycerol-3-phosphate acyltransferase antisense oligonucleotide