If a cancer patient has bone metastases, they will frequently have anemia as well. Given that the bone marrow is the site of blood cell formation, this observation is perhaps not unexpected. But its molecular underpinnings had been unclear to date. Now, researchers at Princeton University and the Rutgers Cancer Institute of New Jersey have identified a specialized group of iron-transporting macrophages in the metastasis-anemia link. Read More
Metabolic dysfunction-associated steatotic liver disease (MASLD) now affects over a third of adults worldwide. Its progressive form, metabolic dysfunction-associated steatohepatitis (MASH), is a leading driver of cirrhosis, cancer and liver transplants while often coexisting with diabetes, obesity and cardiovascular disease. Read More
Aptevo Therapeutics Inc. has unveiled two new trispecific candidates, APVO-452 for prostate cancer and APVO-451 for solid tumors. Powered by the company’s Adaptir-Flex platform, each trispecific immune cell engager biologic brings together tumor targeting, T-cell activation and immune costimulation in a single molecule. Read More
Bright Minds Biosciences Inc. has released preclinical results for BMB‑201, a selective 5-HT2A/2C receptor agonist, in a validated isosorbide dinitrate (ISDN) rat model of vascular headache. BMB-201 is designed to harness the analgesic potential of serotonin modulation without the hallucinogenic effects commonly associated with 5-HT2A activation. Read More
Cytokinetics Inc. has disclosed fast type myosin-binding protein C (MYBPC2) inhibitors reported to be useful for the treatment of neuromuscular disorders. Read More
Biogen Inc.’s IND application for BIIB-142 has been accepted by the FDA. BIIB-142 is a degrader of IRAK-4 that Biogen intends to explore for therapeutic use in patients with autoimmune diseases. Read More
Huntington’s disease is an inherited neurodegenerative disease in which the huntingtin protein undergoes polyglutamine expansion, leading to both loss and gain of functions. A research collaboration spanning France, Germany, the Netherlands and the U.S. showed in previous work that overexpressing CYP46A1 in the brain can mitigate disease in multiple mouse models. Read More
Heme oxygenase 1 (HO-1) is a stress-inducible enzyme that plays a critical role in the pathogenesis of rheumatoid arthritis, a systemic autoimmune disease characterized by chronic synovial inflammation, immune cell infiltration and progressive joint destruction. Read More
Axovia Therapeutics Inc. has been awarded a new $1.0 million grant by the nonprofit organization A Race Against Blindness to support the clinical development of AXV-101, an investigational gene therapy aimed at combating childhood blindness due to retinitis pigmentosa caused by Bardet-Biedl syndrome 1 (BBS1). Read More
Hangzhou Polymed Biopharmaceuticals Inc. has identified proteolysis targeting chimera (PROTAC) compounds comprising a Von Hippel-Lindau disease tumor suppressor (VHL) ligase binding moiety covalently linked to a GTPase KRAS (G12D mutant) targeting moiety via a linker reported to be useful for the treatment of cancer. Read More
Pulmonary arterial hypertension (PAH) is a condition that may lead to right heart dysfunction. Previous evidence has tied mitochondrial dynamics with the progression of PAH, but the mechanisms behind this are not well elucidated. Read More
Congruence Therapeutics Inc. has closed a $32 million financing round to advance its pipeline of small-molecule correctors for diseases of protein misfolding. Read More
Sitala Bio Ltd. has divulged complement factor B (CFB) inhibitors reported to be useful for the treatment of age-related macular degeneration, schizophrenia, atypical hemolytic uremic syndrome, membranous nephropathy, myasthenia gravis, paroxysmal nocturnal hemoglobinuria, diabetic retinopathy and rheumatoid arthritis, among others. Read More
Rapafusyn Pharmaceuticals Inc. has closed a series A financing round raising a total of $44 million to advance its Rapaglues non-degrading molecular glue drug discovery platform and its pipeline in oncology, immunology, renal and pain disease areas. Read More
Lactiga US Inc. has received an award from the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to advance the development of its secretory IgA (sIgA) antibody for inflammatory bowel disease (IBD). Read More