The sex-determining region Y (SRY)-related HMG-box, group E (SOXE) transcription factors SOX9 and SOX10 are essential for the specification and differentiation of many progenitor cell types and for the development of several organs and tissues.
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
Janssen Pharmaceutica NV has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of osteoarthritis, cancer, nonalcoholic steatohepatitis (NASH), skin lesion, sarcoidosis, as well as cardiovascular, metabolic and Alzheimer's diseases, among others.
Silo Pharma Inc. plans to initiate a pilot study of SPU-21, its novel joint-homing peptide targeting rheumatoid arthritis (RA), in human synovial tissue surrounding joints and tendons.
Now, researchers at Stanford University School of Medicine have shown that by targeting CD47 – better known for its role as an innate immune checkpoint akin to PD-1 and CTLA-4 in the adaptive immune system – they were able to restore muscle stem cell function. Aged mice regained muscle strength comparable to younger animals after receiving an antibody treatment targeting CD47 signaling in muscle stem cells.
Gilead Sciences Inc. has divulged thienopyrrole compounds acting as Toll-like receptor 7 (TLR7) and/or TLR8 antagonists reported to be useful for the treatment of systemic lupus erythematosus (SLE), cutaneous lupus erythematosus, lupus nephritis and inflammatory disorders, among others.
Novadip Biosciences SA has raised an additional €40 million in a series B equity round and nondilutive funding to accelerate the clinical development of two of the company's investigational adipose stem cell (ASC)-derived tissue regeneration products.
Dystroglycanopathies are a subset of rare congenital muscular dystrophies (CMDs) caused by dysregulation in the assemblage of glycans on the α-dystroglycan (α-DG) transmembrane glycoprotein.
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