The first cellular human and mouse map focused on muscle fibers and their microenvironment has revealed both the mechanisms of deterioration of this tissue over time and its adaptive capacity for regeneration. “We intended to map the skeletal muscle, isolating all the cell types, and characterizing how they change with age,” first author Veronika Kedlian from the Wellcome Sanger Institute in Cambridge told BioWorld. Read More

Proprotein convertase subtilisin/kexin type-9 (PCSK9) is highly expressed in adult hepatocytes. PCSK9 binds to and promotes the degradation of the low-density lipoprotein (LDL) receptor, thereby increasing LDL cholesterol levels. PCSK9 inhibition has emerged as a promising strategy for cardiovascular diseases. However, it is still unclear whether PCSK9 can trigger blood vessel inflammation directly modulating monocytes or endothelial cells independently of LDL receptor. Read More

Saliogen Therapeutics Inc. has nominated a development candidate, SGT-1001, for the treatment of Stargardt disease. SGT-1001 is being developed as a one-time, nonviral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation. Read More

Derm-Biome Pharmaceuticals Inc. has developed a topical anticancer treatment for patients with or at high risk of developing multiple actinic keratoses. Read More

Researchers from Astrazeneca plc reported preclinical data for AZD-8421, a selective cyclin-dependent kinase 2 (CDK2) inhibitor, currently being evaluated in early-phase clinical trials as a treatment for solid tumors. Read More

Circio Holding ASA has established technical in vivo proof of concept for its proprietary Circvec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression. Read More

Wista Laboratories Ltd. has disclosed thiazole-containing compounds acting as microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of corticobasal syndrome, Alzheimer’s disease, Down syndrome and more. Read More

Bivictrix Therapeutics plc’s BVX-001 has been awarded orphan drug designation by the FDA for acute myeloid leukemia (AML).

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Eli Lilly & Co. has synthesized glucocorticoid receptor (GR) agonists reported to be useful for the treatment of atopic dermatitis and rheumatoid arthritis. Read More

Origenis GmbH has identified cyclin-dependent kinase 9 (CDK9) inhibitors reported to be useful for the treatment of autoimmune disease, cancer, obesity, diabetes, viral infections, cardiovascular disorders and inflammatory disorders. Read More

Unknown etiology is commonly encountered in the kidney pre-transplant routine program. A screening program was performed to detect patients and study recipients that meet the following features: hypertension with no clear etiology and biopsies that do not match with clinical features of classical glomerulopathies. Read More

Shanghai Apeiron Biotechnology Co. Ltd. has divulged protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer. Read More

Pfizer Inc. has described serine/threonine-protein kinase (SIK) inhibitors reported to be useful for the treatment of arthritis, cancer, osteoporosis, atherosclerosis, inflammatory bowel disease, autoimmune diseases, inflammatory disorders and pain. Read More

Poseida Therapeutics Inc. has announced progress with its fully nonviral liver-directed gene therapies. Read More

Despite GTPase KRAS being the most common mutated oncogene in human cancers, there is still an unmet medical need for treating KRAS G12C-mutated cancers. Read More