OrsoBio’s development programs address high unmet medical need in patients with severe metabolic disorders, including type 2 diabetes, severe hypertriglyceridemia, and congenital and acquired lipodystrophies.
Our programs address the root cause of organ dysfunction by modulating fundamental metabolic pathways with four highly targeted and complementary mechanisms:
Type 2 diabetes patients worldwide
Risk of pancreatitis in severe hypertriglyceridemia
Of lipodystrophy patients on approved therapies due to limited efficacy, cost, and/or side effects
Increased fatty acid oxidation in cardiac muscle (ACC2), plus beneficial effects on plasma lipids (LXR, protonophores)
Hepatic DNL inhibition (LXR, protonophores), increased fatty acid oxidation (ACC2, protonophores), increased NAD+ synthesis, and improved mitochondrial function (ACMSD)
Increased NAD+ synthesis and improved mitochondrial function in kidneys (ACMSD)
Increased fatty acid oxidation in skeletal muscle (ACC2)
Each of our programs modulates central aspects of cellular energetics in key, energetically-driven organs
November 1, 2022
Enhanced lipogenesis associated with a high-risk GCKR variant (rs1260326) is effectively suppressed by TLC-2716, a novel, liver-targeted LXR inverse agonist in steatotic human liver organoids (AASLD 2022, Abstract #4210)
November 1, 2022
Evaluation of the safety and pharmacokinetic effects of the oral, acetyl-CoA carboxylase-2 (ACC2) inhibitor TLC-3595 in healthy volunteers (AASLD 2022, Abstract #3677)
November 1, 2022
TLC-6740, a potent liver-targeted mitochondrial protonophore, has multiple metabolic benefits in preclinical models (AASLD 2022, Abstract #2551)
November 1, 2022
TLC-2716, a potent, liver-targeted, inverse agonist of the liver X receptor (LXR), demonstrates profound reductions in hepatic and plasma lipids in dysmetabolic rodent models (AASLD 2022, Abstract #2532)
November 1, 2022
Enhancement of de novo NAD+ biosynthesis by novel ACMSD inhibitors improves mitochondrial function in iPSC-derived human liver organoid models of steatohepatitis (AASLD 2022, Abstract #2441)
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OrsoBio team members collaborated with Professor Takebe in his pioneering study in Cell on the first use of en masse human liver organoids to define the genetic basis of metabolic liver disease and potential for tailored therapeutic development.