PARP inhibitors such as olaparib are used for the treatment of metastatic castration-resistant prostate cancer (mCRPC) harboring homologous recombination deficiency (HRD), but a proportion of these patients do not respond to therapy or eventually develop resistance.
Arthex Biotech SL has received FDA clearance to initiate the phase I/IIa Arthemir study of ATX-01 for the treatment of myotonic dystrophy type 1 (DM1).
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.
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