The human genome, the sequence that represents the DNA of our species, was built with a single individual as a model. This all-in-one standard didn’t include the gene variations that make us different or explain why some people develop certain diseases. Four simultaneous studies from the Human Pangenome Reference Consortium have published a sequence based on 47 individuals, beginning to capture the genetic diversity that defines humans. Read More
The aqueous supernatants resulting from ultracentrifugation of brain samples from patients with Alzheimer’s disease (AD) contain aggregates so far described as soluble oligomers of amyloid-β protein (Aβ), which are responsible for the neurotoxicity underlying AD and thus considered targets to watch in this devastating condition. Now, a group of scientists from Harvard Medical School have determined that these aggregates are in fact insoluble diffusible fibrils with the same atomic structure as plaque fibrils. Read More
Rocket Pharmaceuticals Inc. has received IND clearance from the FDA for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM). RP-A601 offers the potential for a one-time, curative alternative to medical therapy, implantable cardioverter defibrillators and ablations. Read More
Research at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. has led to the identification of sulfonylpiperazinyl compounds acting as UDP-2,3-diacylglucosamine hydrolase (LpxH) inhibitors and reported to be useful for the treatment of gram-negative bacterial infections. Read More
Cell cycle dysregulation in cancer cells represents a clinical option for treating cancer, by using cyclin-dependent kinase 4/6 (CDK4/6) inhibitory agents. Further evaluation on this approach was presented by TYK Medicines Inc., where they focused on the pharmacological action of a CDK7 inhibitor TY-2699a for the potential treatment of cancer. Read More
Amgen Inc. and Tscan Therapeutics Inc. have entered into a multi-year collaboration that will use Tscan’s proprietary target discovery platform, Targetscan, to identify the antigens recognized by T cells in patients with Crohn’s disease. Read More
Beijing Tide Pharmaceutical Co. Ltd. has prepared proteolysis-targeting chimera (PROTAC) compounds comprising an ubiquitin ligase binding moiety bound to a protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) targeting moiety through a linker. Read More
Researchers from Kanaph Therapeutics Inc. have reported the discovery and preclinical evaluation of a small-molecule EGFR inhibitor, KNP-501, being developed as an anticancer agent. Read More
Researchers from Hibercell Inc. presented preclinical data for the eukaryotic translation initiation factor 2-α kinase 3 (PERK) inhibitor HC-5404, currently in phase I development for the treatment of solid tumors (NCT04834778). Read More
Pyrimidopyridine derivatives acting as GTPase KRAS G12D inhibitors have been reported in a Sunshine Lake Pharma Co. Ltd. patent as potentially useful for the treatment of cancer. Read More
Additional early-stage research and drug discovery news in brief, from: Innocan Pharma, Palatin Technologies, Sana Biotechnology, Seismic Therapeutic. Read More
