Solid Biosciences Inc. has announced approvals of its IND application and CTA by the U.S. FDA and Health Canada, respectively, for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by mutations in the RYR2 and CASQ2 genes.
Human adenoviruses (HAdVs) are nonenveloped viruses with linear double-stranded DNA. Though often self-limiting, they can cause severe disease in immunocompromised individuals. The compact genome of HAdV results in few viable drug targets, complicating efforts to develop new treatments. Consequently, antiviral options remain limited, with most candidates being nucleoside or nucleotide analogues.
Researchers at the Chinese Academy of Sciences and collaborators have developed a new method to label and monitor dormant breast cancer cells over time, shedding light on how these cells survive chemotherapy and potentially trigger metastatic relapse in the lung. Breast cancer frequently recurs in distant metastatic sites, even after the primary tumor has fully regressed following initial therapy.
Additional early-stage research and drug discovery news in brief, from: Artelo Biosciences, Cyclacel Pharmaceuticals, Hanmi Pharmaceuticals, Hotspot Therapeutics.
Aussie researchers have used CRISPR gene editing tools to “armor” chimeric antigen receptor (CAR) T cells to activate additional cancer-fighting proteins at the tumor site, enabling them to target cancer cells in solid tumors.
Acurex Biosciences Corp. has divulged fused quinone compounds acting as ferroptosis inhibitors reported to be useful for the treatment of mitochondrial disease.
Deciphera Pharmaceuticals LLC has identified phosphatidylinositol 3-kinase catalytic subunit type 3 (PIK3C3; Vps34) inhibitors reported to be useful for the treatment of cancer.
Alterome Therapeutics Inc. has synthesized RAC-α serine/threonine-protein kinase (AKT1; PKB α) (E17K mutant) inhibitors reported to be useful for the treatment of cancer.
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