Aicardi Goutières Syndrome (AGS)
https://www.immunesensor.com/news/021226
ImmuneSensor Therapeutics recently successfully completed a control Phase 1a study in healthy volunteers to evaluate the safety, tolerability, pharmacokinetics (what the body does to the drug) and pharmacodynamics (what the drug does to the body) of their product IMSB301 a novel, potentially best-in-class, orally available small molecule cGAS inhibitor. It was well tolerated and helped inform the best and safest dose for the Phase 1b clinical trial that is expected to enrol up to six patients with ultra-rare Aicardi Goutières Syndrome (AGS) and interferonopathies. They have now dosed their first AGS patient with IMSB301 in this clinical trial in Australia and hope to have some results later this year. Positive results would provide the opportunity to advance this drug for AGS, taking advantage of the Orphan Drug Designation, it received from the U.S. Food and Drug Administration (FDA) which helps with the development of drugs for rare diseases.