It has been previously demonstrated that in zebrafish, the activity of UXS1, a gene that encodes UDP-glucuronate decarboxylase 1, is essential for production and organization of skeletal extracellular matrix. Now, researchers from the University of Oslo and affiliated organizations have identified a novel pathogenic variant in UXS1.
Gene therapy developer Kate Therapeutics Inc. (KateTx), which is developing next-generation adeno-associated virus (AAV) vectors that target skeletal and cardiac muscle, has unveiled $51 million series A round and a licensing deal with Astellas Pharma Inc.
CD38 is an enzyme with NAD-depleting and intracellular signaling activity expressed on the cell surface, in intracellular compartments and in mitochondria, and is linked to inflammatory and autoimmune diseases. Studies in CD38-deficient mice have revealed that these animals develop a type of collagen-induce arthritis (CIA) which suggests a link between CD38 and CIA pathogenesis.
Aria Pharmaceuticals Inc. has described C-C chemokine receptor type 2 (CCR2; MCP-1-R) modulators reported to be useful for the treatment of systemic lupus erythematosus (SLE) and lupus nephritis.
Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
Exo Therapeutics Inc. has announced its lead program directed against TANK-binding kinase 1 (TBK1) for the treatment of autoimmune diseases. The company is now approaching identification of a development candidate for its lead program, an exosite-targeted compound that selectively reprograms the activity of TBK1 in the STING pathway that drives pathogenic signaling in diseases such as systemic lupus erythematosus, Aicardi-Goutières syndrome and others.
RSS
