Abbvie Inc. recently disclosed the discovery and structure of the anti-tumor necrosis factor (TNF) glucocorticoid receptor modulator (GRM) immunology antibody-drug conjugate (iADC) ABBV-154. The drug is in phase II clinical development as a subcutaneous treatment for rheumatoid arthritis and polymyalgia rheumatica, and as a subcutaneous or intravenous treatment for active Crohn’s disease.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy. Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
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, cutaneous lupus erythematosus, lupus nephritis and inflammatory disorders, among others.
Shares of Avidity Biosciences Inc. (NASDAQ:RNA) dropped 15% March 30 as the company disclosed details of the serious adverse event (SAE) that prompted a partial clinical hold in September 2023 on the phase I/II trial testing AOC-1001 in adults with myotonic dystrophy type 1 (DM1).
Autophagy plays a critical role in the regulation of skeletal muscle integrity; however, the molecular mechanisms regulating autophagy are not fully understood. In a recently published study, researchers from McGill University Health Centre and affiliated organizations aimed to investigate these mechanisms and identify novel regulators of autophagy and skeletal muscle integrity.
Restoring levels of the immune modulator mesencephalic astrocyte-derived neurotrophic factor (MANF) in the muscles of aged animals improved their regenerative capacity by promoting repair-associated injury responses, researchers from the University of Lisbon have discovered.
Partially blocking a receptor that helps regulate the activity of the inflammatory cytokine molecule interleukin-6 (IL-6) seems to promote tissue regeneration and block degeneration in a model of osteoarthritis. As reported in the March 22, 2023, issue of Science Translational Medicine, the receptor, called glycoprotein 130 (gp130), regulates both positive and negative inflammatory responses that can help regenerate tissue, but also cause degeneration.
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