Interleukin-1 receptor-associated kinase 4 (IRAK-4) is a modulator of IL-1 receptor and Toll-like receptor (TLR) signaling and has emerged as a promising therapeutic target for several inflammatory diseases. Gilead Sciences Inc. and Nurix Therapeutics Inc. recently presented data generated in a preclinical murine model of AD with their IRAK-4 degrader GS-6791.
In heart failure, the heart does not oxygenate body tissues adequately. A standard treatment is SGLT2 inhibitors such as empagliflozin and dapagliflozin, derived from the national product phlorizin. However, long-term use of these inhibitors increases risk of renal damage and euglycemic diabetic ketoacidosis.
Chineses researchers investigated the relationship between formyl peptide receptor 1 (FPR1) expression and neurodegeneration in multiple sclerosis (MS), specifically evaluating the therapeutic potential of the FPR1 antagonist T-0080.
Biolinerx Ltd. and Hemispherian AS have established a joint venture (JV) to develop GLIX-1, Hemispherian’s lead drug candidate being developed as a potential treatment for newly diagnosed and recurrent glioblastoma.
Climb Bio Inc. has announced results from a completed nonhuman primate (NHP) study of CLYM-116, an anti-APRIL monoclonal antibody (mAb) in development for IgA nephropathy.
Orexo AB’s subsidiary Orexo US Inc. has been awarded $8 million in funding by the U.S. Biomedical Advanced Research and Development Authority (BARDA) to support the development of OX-390, an intranasal rescue medication for adulterated opioid overdoses.
Tevard Biosciences Inc. has presented new preclinical data on the use of therapeutic suppressor tRNAs (suptRNAs) for the treatment of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM). The data show potent restoration of full-length functional proteins in models of DMD and DCM caused by titin truncations (DCM-TTNtv).
Two independent studies applied CRISPR-based genetic editing – one to treat leukemia and the other to target myeloma – to overcome the challenges faced by CAR T cells, such as exhaustion, impaired activation and fratricide, a phenomenon in which they attack each other.
Idorsia Pharmaceuticals Ltd. has divulged macrocyclic compounds acting as cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.