Otoferlin is a calcium sensor protein critical for the transmission of the signal from inner hair cells (IHCs) to the spiral ganglion neurons (SGNs), and it is encoded by the OTOF gene. Pathogenic biallelic loss of function variations in OTOF result in failure of synaptic transmission, causing autosomal recessive deafness 9 (DFNB9), which is a congenital severe-to-profound auditory neuropathy.
Assembly Biosciences Inc. has selected development candidate ABI-5366 to progress to IND-enabling studies from its long-acting herpes simplex virus type 2 (HSV-2) helicase inhibitor program for high-recurrence genital herpes. The company is targeting IND/CTA filings in the first half of next year.
Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. has presented protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer.
Jiangsu Hengrui Medicine Co. Ltd., Shanghai Hengrui Pharmaceutical Co. Ltd., Shanghai Senhui Pharmaceutical Co. Ltd. and Shanghai Shengdi Pharmaceutical Co. Ltd. have identified antibody-drug conjugates (ADCs) comprising HER2-targeting antibody or antigen-binding fragments linked to eribulin derivatives through a cleavable linker reported to be useful for the treatment of cancer.
Tuojie Biotech (Shanghai) Co. Ltd. has described lysophosphatidic acid receptor 1 (LPAR1) antagonists reported to be useful for the treatment of metabolic diseases, cancer, liver diseases, respiratory, renal, neurological, cardiovascular and eye disorders, among others.
Micurx Pharmaceuticals Inc. has patented peptide-drug conjugates consisting of peptide targeting kidney cells covalently linked to an anti-inflammatory or immunomodulating or nephron-protective drug through a linker reported to be useful for the treatment of acute kidney injury, chronic kidney disease, chronic glomerulonephritis, diabetic nephropathy and systemic lupus erythematosus, among others.
Microbio (Shanghai) Co. Ltd. has synthesized glucagon-like peptide 1 receptor (GLP1R) agonists reported to be useful for the treatment of obesity, type 2 diabetes and nonalcoholic fatty liver disease.
Cathepsin L (CTS-L) is a lysosomal enzyme responsible for degrading endocytosed proteins to generate immunogenic antigens for adaptive immunities. In the current study, researchers reported data from a study that aimed to assess the potential of targeting procathepsin L (pCTS-L) as a potential therapeutic strategy against sepsis.
Triple-negative breast cancer (TNBC) still remains as one of the most aggressive breast cancer types among women, with high metastatic potential and ability to shift towards epithelial-mesenchymal transition (EMT).
Scientists from the National Institutes of Health and affiliated organizations established a novel mouse model of intraventricular extension of hemorrhage (IVH) for the purpose of measuring motor and cognitive function in acute and chronic stages of intracerebral hemorrhage, which is the most severe stroke subtype.
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