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.
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.
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.
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).
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.
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.
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.
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.
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has patented proteolysis targeting chimera (PROTAC) compounds comprising cereblon (CRBN) ligands covalently bonded to a Bcl-2-like protein 1 (Bcl-xl; Bcl-X; BCL2L1)-targeting moiety through a linker.
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