Gilead Sciences Inc. has exercised its option to exclusively license Nurix Therapeutics Inc.'s investigational targeted protein degrader molecule NX-0479, now designated GS-6791. This bivalent degrader is the first development candidate resulting from the companies' collaboration to discover, develop and commercialize up to five innovative targeted protein degradation therapies.
Systemic lupus erythematosus (SLE) is an autoimmune disease characterized by disrupted tolerance against nucleic acids, which form immune complexes with antibodies, finally leading to tissue damage. However, the mechanisms underlying the release of DNA from cells remain unexplained.
Osteoarthritis and its associated cartilage pathology affects 30 million people in the U.S., but no disease-modifying treatments have yet reached the clinic. A recent multicenter trial evaluating the safety and efficacy of a truncated, recombinant human fibroblast growth factor-18 (FGF18) protein analogue (rhFGF18) demonstrated a dose-dependent improvement in cartilage thickness relative to a placebo.
Researchers have linked Duchenne muscular dystrophy (DMD) to a loss of regenerative capacity of muscle stem cells. The findings, which were published in the March 1, 2023, issue of Science Translational Medicine, suggest that boosting the regenerative capacity of muscle stem cells could delay or perhaps even prevent the progression of DMD. DMD is “an early and horrible disease,” senior author Frederic Relaix, who is the director of a research team studying the biology of the neuromuscular system at the Mondor Institute for Biomedical Research told BioWorld.
Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
Genascence Corp. has been awarded US$11.6 million over 4 years from the California Institute for Regenerative Medicine (CIRM) to help advance the company's GNSC-001 gene therapy for knee osteoarthritis (OA). The funding will support a phase Ib trial and manufacturing activities.
Using single-cell RNA sequencing of deer antler at different stages of their annual cycle of regeneration, Chinese researchers have identified a progenitor cell population that drove antler regeneration. The authors of an accompanying editorial wrote the findings, which were published in the Feb. 24, 2023, issue of Science, “add to the emerging idea that blastema progenitor cells are a common stem cell type in mammalian appendage regeneration.”
Antibodies against tumor necrosis factor (TNF) and IL-6 receptor are the most effective available therapies against rheumatoid arthritis (RA). However, no cure for RA exists. In work published in Science Translational Medicine, scientists from Sanofi R&D (Sanofi SA) investigated whether dual inhibition of TNF and IL-6 could improve the therapeutic efficacy against RA.
Hebei Feinisi Biotechnology Co. Ltd. has synthesized shared epitope-containing HLA DR molecule (SE-DR) affinity peptides reported to be useful for the treatment of rheumatic diseases.
Bone-resorbing osteoclasts are key players in bone remodeling and their dysfunction has been implicated in several bone disorders such as fibrous dysplasia. Osteoclasts derive from monocytes stimulated by several cytokines, such as M-CSF and RANKL.
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