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.
Blueprint Medicines Corp. scored a broader label from the U.S. FDA for Ayvakit (avapritinib), which became the first approved therapy to treat adults with indolent systemic mastocytosis (ISM).
Sarepta Therapeutics Inc.’s balloting March 12 from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (OTAT) in favor of gene transfer therapy SRP-9001 (delandistrogene moxeparvovec) in Duchenne muscular dystrophy (DMD) had Wall Street mulling the odds for others in the space.
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