CRISPR, or clustered regularly interspaced palindromic repeats, is transforming biomedical research, and making rapid inroads into the clinic, with its ability to easily target specific DNA and RNA sequences. CRISPR itself is made of RNA. It recognizes target sequences and delivers CRISPR-associated (Cas) proteins, nucleases that cut the target sequence. In two papers published online in Nature on Jan. 4, 2023, researchers have demonstrated that a recently discovered type of Cas protein, Cas12a2, can degrade double-stranded DNA when its associated CRISPR guide RNA recognizes its target sequence. Read More
After comparing the response to the two types of vaccines for the respiratory syncytial virus (RSV) based on its fusion protein (F), prefusion (pre-F) versus postfusion (post-F) vaccines, scientists at the National Institutes of Health (NIH) and Astrazeneca plc have demonstrated that targeting the pre-F protein led to better protection. No more bets on RSV immunization based on the post-F protein of the virus. Laboratories can now bet all on red for the pre-F technology. Read More
A model of tauopathy developed in the worm nematode Caenorhabditis elegans was developed for investigating the mechanisms behind tauopathy development. Read More
Shanghai Raising Pharmaceutical Co. Ltd. has disclosed pyrimidines acting as amyloid-β (Aβ) protein production inhibitors reported to be useful for the treatment of Alzheimer's disease. Read More
CSPC Pharmaceutical Group Ltd. has received clearance from China's National Medical Products Administration (NMPA) to conduct clinical trials in China of the highly selective novel protein arginine methyltransferase 5 (PRMT5) inhibitor SYH-2045 for the treatment of advanced malignant tumors. Read More
Multiple myeloma (MM) represents about 10% of all blood cancers, remaining an incurable disease with a 5-year overall survival rate of 56%. B-cell maturation antigen (BCMA) is a receptor in the cell surface that is highly expressed in malignant plasma cells, and in normal cells, that promotes cell proliferation and survival by binding to APRIL and BAFF ligands. Read More
Ventus Therapeutics Inc. has nominated a potential first-in-class cyclic GMP-AMP synthase (cGAS) inhibitor, VENT-03, as the company’s first development candidate directed against cGAS. Read More
Johns Hopkins University has described prodrugs of sphingomyelin phosphodiesterase 3 (SMPD3; nSMase2) inhibitors reported to be useful for the treatment of cancer, neurological disorders, infections and inflammatory disorders. Read More
Cancer Focus Fund LP is investing US$5 million in funding to support ISA Pharmaceuticals BV's ISA-103 in a first-in-human study for the treatment of uveal melanoma. Read More
Metacen Therapeutics Corp. has synthesized compounds able to capture methylglyoxal (MGO) reported to be useful for the treatment of aging, diabetes, hyperlipidemia, neurodegeneration, autism spectrum disorder, nonalcoholic fatty liver disease (NAFLD), fibrosis and cardiovascular disorders. Read More
SFA Therapeutics Inc. has received FDA clearance of its IND application to investigate SFA-001N in patients with nonalcoholic steatohepatitis (NASH) with or without fibrosis. Read More
Tencent Technology (Shenzhen) Co. Ltd. has disclosed pyrrolo[2,1-f][1,2,4]triazine derivatives acting as dual specificity protein kinase TTK inhibitors reported to be useful for the treatment of cancer. Read More
