At the 2024 European College of Neuropsychopharmacology (ECNP) Congress, researchers have presented work that could lead to ways to boost brain development and prevent neurodegeneration in individuals with Down syndrome. Read More
In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, GENE-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids. Read More
Researchers from Moderna Inc. and affiliated organizations presented preclinical data for the novel mRNA-lipid nanoparticle (LNP) vaccine, mRNA-1769, against mpox virus. Read More
The FDA has awarded U.S. orphan drug designation to Eydisbio Inc.’s EYD-001 (formerly HS-276), a highly selective and potent, orally bioavailable TAK1 inhibitor for the treatment of systemic sclerosis. Eydisbio plans to initiate clinical trials in the near future. Read More
To date, therapies for multiple sclerosis (MS) focus on modifying or suppressing the immune system rather than on remyelination. Recent findings have pointed to the κ-opioid receptor (KOR) as a therapeutic target for remyelination, but several KOR agonists have undesired side effects that limit their use. Researchers from the Victoria University of Wellington have tested KOR agonists derived from U-50488 in the preclinical setting for the management of MS. Read More
Uveitis is a disease involving the eye, affecting the iris, ciliary body, retina and their associated blood vessels, among others. The non-infectious type of uveitis is linked to immune-related factors and its pathogenesis remains poorly understood. Read More
Researchers at the University North Carolina at Chapel Hill and University of Florida have divulged triazoles acting as κ-opioid receptor agonists reported to be useful for the treatment of pain, pruritus, depression, inflammation and more. Read More
High extracellular glutamate levels damage axons, myelin and oligodendrocytes in the context of inflammatory demyelinating disorders such as multiple sclerosis (MS). Read More
Vanderbilt University has synthesized metabotropic glutamate (mGlu2) receptor negative allosteric modulators reported to be useful for the treatment of depression, anxiety, Alzheimer's disease, autism spectrum, obsessive compulsive and cognitive disorders. Read More
Merck Sharp & Dohme LLC and Otsuka Pharmaceutical Co. Ltd. have patented cellular tumor antigen p53 (TP53) (Y220C mutant) activators reported to be useful for the treatment of cancer. Read More
Zic family member 4 (ZIC4) is a tumor suppressive transcription factor that is epigenetically silenced in many types of cancers. In the current study, researchers from Children's Cancer Hospital Egypt (CCHE) and affiliated organizations investigated the epigenetic regulation function of ZIC4 in pediatric choroid plexus tumors (CPTs). Read More
Gan & Lee Pharmaceuticals Co. Ltd. has described new S-adenosylmethionine synthase isoform type-2 (Mat2A) inhibitors reported to be useful for the treatment of cancer. Read More
The differentiation of oligodendrocyte progenitor cells (OPCs) into oligodendrocytes (OLs) promotes the remyelination in human brain. In multiple sclerosis (MS), dysfunctional OPC differentiation leads to remyelination failure and subsequent severe neurological disability. Read More
Palisade Bio Inc. has submitted a clinical trial application (CTA) for PALI-2108 for the treatment of ulcerative colitis, following a pre-CTA consultation meeting with Health Canada. Pending clearance, the company anticipates initiating a phase I study by year-end. Read More
The University of Michigan has identified proteolysis targeting chimeric (PROTACs) compounds comprising cereblon (CRBN) ligands covalently bonded to a CREB-binding protein (CREBBP; CBP) and/or histone acetyltransferase KAT3B (p300)-targeting moiety through a linker. They are described as potentially useful for the treatment of cancer, autoimmune and inflammatory disorders. Read More
