The discovery of a complex formed by two types of ion channels in neurons has allowed researchers from Heidelberg University to develop an inhibitor that stopped motor neuron degeneration in amyotrophic lateral sclerosis (ALS) in mouse models and human brain organoids. Read More
Researchers from JCR Pharmaceuticals Co. Ltd. have presented new data for JR-171, a novel enzyme replacement therapy currently in early clinical development for the treatment of mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome. Read More
Auron Therapeutics Inc. has nominated its first development candidate, AUTX-703, which is being developed for the treatment of patients with small-cell lung cancer, neuroendocrine prostate cancer and acute myeloid leukemia. Read More
Eli Lilly & Co. has selected FHD-909, a first-in-class oral BRM-selective inhibitor, for clinical development from its 2021 collaboration with Foghorn Therapeutics Inc. Lilly plans to file an IND for FHD-909 in the second quarter of this year. The primary target patient population is BRG1-mutated non-small-cell lung cancer. Read More
At this week’s WORLDSymposium meeting, researchers from M6P Therapeutics Inc. reported on the preclinical efficacy of M-021, a novel enzyme replacement therapy (ERT) that co-expresses recombinant GAA with a bicistronic vector encoding N-acetylglucosamine-1-phosphotransferase (PTase; S1-S3). Read More
Noise-induced hearing loss is still the most common cause of acquired hearing loss nowadays. The mechanisms behind this may be explained through inflammatory responses in the cochlea after acoustic trauma. Read More
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have described new 5’-nucleotidase (CD73) inhibitors reported to be useful for the treatment of cancer. Read More
The main causes of vascular cognitive impairment and dementia (VCID) are the breakdown of the blood-brain barrier, reactive astrogliosis and loss of myelination, but its molecular and cellular mechanisms are not clear enough. Read More
Protein arginine N-methyltransferase 5 (PRMT5) inhibitors have been disclosed in an Abbisko Therapeutics Co. Ltd. patent and described as potentially useful for the treatment of cancer. Read More
Work at Jnana Therapeutics Inc. has led to the identification of sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatohepatitis (MASH), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism spectrum disorders, among others. Read More
Mitochondrial DNA (mtDNA) is comprised of two strands, the light and the heavy one. The latter is rich in guanine and tends to form G-quadruplexes (mtG4s) which are more prevalent in cancer cells and are considered an emerging therapeutic target in cancer treatment. Read More
Incyte Corp. has developed fused tricyclic compounds acting as GTPase KRAS (mutant) inhibitors. As such, they are reported to be useful for the treatment of cancer, inflammation and immunological disorders. Read More
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have patented phosphorylpurinone compounds acting as Toll-like receptor 7 (TLR7) agonists reported to be useful for the treatment of cancer. Read More
Researchers from National Taiwan University combined the structural features of purine-type microtubule-disrupting compounds and histone deacetylase (HDAC) inhibitors, which led to the discovery of a novel series of dual-targeting compounds, with the purine-hydroxamate conjugate [I] selected as the lead candidate. Read More
