Wigen Biomedicine Technology (Shanghai) Co. Ltd. has divulged pyrrolopyrimidine derivatives containing a pyrazine structure acting as Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer.
Opna Bio AG has identified transcriptional coactivator YAP1/transcriptional enhancer factor (TEAD) interaction inhibitors reported to be useful for the treatment of arrhythmogenic right ventricular cardiomyopathy, cancer, Holt-Oram syndrome, neurofibromatosis type 2, polycystic kidney and Alzheimer's disease.
Sionna Therapeutics Inc. has received FDA clearance of its IND application for SION-638, a small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A phase I study is now dosing healthy volunteers.
The archetypal UbiB protein COQ8 has human homologues COQ8A and COQ8B, both with well-established connection to human disease, with inactivating mutations in COQ8A resulting in autosomal recessive cerebellar ataxia. Researchers from the University of Wisconsin-Madison and affiliated organizations have now recently reported the discovery of small-molecule inhibitors of COQ8A.
BeadTech Inc. and Sogang University have synthesized compounds using pyrrolidine as a linker reported to be useful for the treatment of eczema, psoriasis, pruritus, contact and atopic dermatitis.
Vertex Pharmaceuticals Inc. and Entrada Therapeutics Inc. have entered into a global collaboration focused on discovering and developing intracellular Endosomal Escape Vehicle (EEV) therapeutics for myotonic dystrophy type 1 (DM1).
Zhejiang Hisun Pharmaceutical Co. Ltd. and Shanghai Aryl Pharmtech Co. Ltd. have disclosed bicyclic heteroaryl derivatives acting as tyrosine-protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) allosteric inhibitors reported to be useful for the treatment of cancer, Noonan syndrome, LEOPARD syndrome, fibrosis, cardiovascular and immunological disorders.
CSPC Pharmaceutical Group Ltd. has obtained clearance from China's National Medical Products Administration (NMPA) to conduct clinical trials in China with SYH-2055, an oral small-molecule 3C-like protease (3CLpro) inhibitor against SARS-CoV-2.
A strong clinical association has been observed between epilepsy and the development of depression, however, it is difficult to study individual molecular and cellular mechanisms underlying these comorbidities in animal models. Researchers from Rutgers University have aimed to develop a cell-type-specific monogenic mouse model of epilepsy and depression comorbidities, which could serve as a tool for identifying disease mechanisms as well as for target and drug screening.
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