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.
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.
Newco Relation Therapeutics Ltd. is showing its colors after raising $25 million in a seed round to work on integrating single cell transcriptomics, functional genomics and machine learning – and cut through previously undecipherable combinatorial space – to find and validate drug targets in the non-coding genome.
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.
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