Researchers from Macquarie University have detailed the discovery of a novel gene therapy vector targeting pathological TAR-binding protein 43 (TDP-43), CTx-1000, as a potential therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) – two diseases characterized by cytoplasmic deposition of the nuclear TDP-43.
Derm-Biome Pharmaceuticals Inc. is preparing to initiate IND-enabling studies with DB-007-4, a first-line topical treatment for inflammatory skin diseases, such as acne, atopic dermatitis and rosacea. The company will shortly begin a GLP toxicology study.
Mutations in both KRAS and BRAF oncogenes, which are frequently found in colorectal cancer, are associated with poor prognosis and treatment resistance.
Researchers have identified NIMA-related kinase 1 (NEK1) as a potential therapeutic target driving tumor growth. It was identified using Turbine Ltd.’s Simulated Cell platform when they simulated perturbations in the DNA damage response pathways. NEK1 is involved in DNA damage response, cell cycle and mitosis.
Researchers from Nanjing Medical University presented data from a study that aimed to investigate the role of amphiregulin (AREG) in activating intestinal fibroblasts and driving fibrogenesis.
Henan Medinno Pharmaceutical Technology Co. Ltd. has synthesized TGF-β receptor type-1 (TGFBR1; ALK5; SKR4; TβR-I) inhibitors reported to be useful for the treatment of cancer, aging, arteriosclerosis, diabetic nephropathy, Alzheimer’s disease, viral infections, osteoporosis and skin fibrosis, among others.
Blacksmith Medicines Inc. has disclosed UDP-3-O-(R-3-hydroxymyristoyl)-N-acetylglucosamine deacetylase (LpxC) (bacterial) inhibitors reported to be useful for the treatment of Pseudomonas aeruginosa infection.
Researchers recently conducted a study to identify small molecules slowing metabolism and mimicking states normally induced by hypothermia or hibernation. The final aim was the discovery of drugs to preserve living cells, tissues and organs ex vivo, and potentially in vivo.
Baseimmune Ltd. has closed $11.3 million (£9 million) in series A funding. The company uses proprietary, deep learning artificial intelligence (AI) to predict future pathogen mutations to generate novel vaccines.
Max Biopharma Inc. and Metaba LLC are collaborating to study the effects of oxysterol drug candidates that target metabolic dysfunction-associated steatohepatitis (MASH, formerly nonalcoholic steatohepatitis or NASH), idiopathic pulmonary fibrosis, chronic inflammation, and atherosclerosis on metabolic processes.
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