Tofersen’s development is progressing in fits and starts. That was evident at the U.S. FDA’s March 22 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee, which unanimously agreed Biogen Inc.’s failed phase III study predicted a clinical benefit in treating amyotophic lateral sclerosis (ALS) that includes the rare superoxide dismutase 1 component.
Courage Therapeutics Inc. and University of Michigan have synthesized peptides acting as melanocortin MC4 receptor (MC4R) agonists and/or MC3 receptor (MC3R) antagonist/partial agonists reported to be useful for the treatment of arthritis, depression, diabetes, hypertension, renal disorders and sleep apnea.
A research team led by Domagoj Cikes at the Institute of Molecular Biotechnology of the Austrian Academy of Sciences (IMBA) and Josef Penninger at the University of British Columbia has discovered the critical role of the enzyme phosphate cytidylyltransferase 2 (PCYT2) in muscle health. Their findings appeared in the March 20, 2023, online edition of Nature Metabolism.
Shanghai Visonpharma Co. Ltd. has described Toll-like receptor 7 (TLR7) and/or TLR8 antagonists reported to be useful for the treatment of multiple sclerosis, lupus nephritis, psoriasis, rheumatoid arthritis, systemic scleroderma (systemic sclerosis), Sjogren syndrome and systemic lupus erythematosus.
Researchers from the University of Utah applied RNASeq analysis for an undiagnosed case of a critically ill newborn with a complex phenotype, with the aim of providing better diagnosis and improving treatment outcomes.
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.
Despite a failed phase III study, the U.S. FDA suggests in briefing documents that tofersen (BIIB-067) is effective for treating the rare, genetic disease superoxide dismutase 1 amyotrophic lateral sclerosis (ALS). The intrathecally injected therapy is being developing by Biogen Inc. and Ionis Pharmaceuticals Inc. and is at the heart of a March 22 meeting of the agency’s Peripheral and Central Nervous System Drugs Advisory Committee (adcom).
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.
Quralis Inc. raised $88 million in series B round to fund clinical development of its two lead programs in amyotrophic lateral sclerosis (ALS) and to take forward earlier-stage pipeline projects in ALS and frontotemporal dementia.
RSS
