Iqure Pharma Inc. has received institutional review board (IRB) approval for a first-in-human study of iQ-007 in healthy volunteers. This follows recent formal approval from the Human Research Ethics Committee (HREC) in Australia for the phase I study, which is expected to begin next month.
The Ca2+ stored in the cellular endoplasmic reticulum (ER) plays a crucial role in protein folding and lipid transfer, and its impairment leads to cellular ER stress. When chronic cellular ER stress occurs in the liver, it triggers the pathogenesis of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH). Previous reports found that NACHT and WD repeat domain containing 1 (NWD1) localized in the ER and mitochondria in neural stem/progenitor cells, but the significance of NWD1 outside the brain is not well known.
Although safe and effective vaccines for SARS-CoV-2 have been successfully developed, there are currently no therapeutic approaches available for treating acute infection, particularly for individuals at high risk of severe disease progression, and for preparedness against a potential new coronavirus pandemic.
Maxion Therapeutics Ltd. has raised $72 million (£58 million) in a series A financing to support its development of antibody-based Knotbody drugs for ion channel- and G protein-coupled receptor (GPCR)-driven diseases.
Neuroblastoma, an aggressive malignancy originating from neural crest cells, accounts for 15% of cancer-related deaths in children. Treatment strategies include systemic chemotherapy, radiation or immunotherapy with anti-GD2 antibodies, all with severe side effects and long-term toxicity. Retinoic acid (RA) has been shown to promote neuroblastoma growth inhibition while suppressing MYCN oncogene expression. However, its effect is reversible, and tumor regrowth may occur.
A combination of Epstein-Barr virus (EBV) antibodies and genetic factors may be linked to an increased risk of multiple sclerosis (MS), according to a study led by scientists at Karolinska Institutet and Stanford University. “The Epstein-Barr virus has been a suspect for many years for having a role in causing MS. The evidence for it has increased though one has not really reached complete proof of its role,” Tomas Olsson told BioWorld.
Onkure Therapeutics Inc. has described phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of cancer, congenital lipomatous overgrowth, vascular malformations, epidermal naevi and skeletal abnormalities, scoliosis and PIK3CA related overgrowth spectrum (PROS), among others.
Biogen Inc. has divulged 3-β-hydroxysteroid-δ(8),δ(7)-isomerase (EBP) inhibitors reported to be useful for the treatment of autoimmune disease and multiple sclerosis.
Selene Therapeutics Ltd. has identified brain permeable dihydroorotate dehydrogenase (DHODH) inhibitors reported to be useful for the treatment of epilepsy, cancer, Alzheimer’s disease, rheumatoid arthritis and multiple sclerosis.
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