While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

Nilo Therapeutics Inc. has launched with a $101 million series A financing and a focus on harnessing neural circuits to restore immune homeostasis in disease.

Researchers at Hefei Amvite Pharmaceutical Co. Ltd. reported the discovery and preclinical evaluation of a series of structurally modified ziritaxestat derivatives, leading to the identification of more potent autotaxin inhibitors.

Researchers from Onco3r Therapeutics BV presented preclinical data on O3R-5671, a novel salt-inducible kinase 3 (SIK3) inhibitor developed for the treatment of inflammatory bowel disease and other autoimmune diseases.

Activation of cannabinoid receptors in the peripheral nervous system may help treat inflammatory and gastrointestinal disorders as well as pain, and several full agonists have been reported but they present safety concerns. Partial agonists, such as those that activate receptors to 20%-50% of full activity, may be safer, yet so far such agonists have emerged serendipitously.