Two STING agonists for COVID-19; plus targeted gene therapy delivery for infantile parkinsonism, Verve’s  hypercholesterolemia program and more

Researchers from the University of Massachusetts and the University of Pennsylvania separately published in Science Immunology studies that suggest small molecule agonists of STING could serve as COVID-19 therapeutics. The UMass team demonstrated that intranasal delivery of diABZI-4, given before or 12 hours post-infection, improved survival and reduced weight loss in mice infected with SARS-CoV-2. In the UPenn study, pretreatment with intranasal diABZI similarly boosted survival and decreased weight loss in infected mice. Intranasal diABZI also showed efficacy against the South African variant, with treated mice exhibiting reduced weight loss and viral lung loads.

Stereotactic targeting could reduce neurotoxic side effects associated with gene therapies for neonatal neurodegenerative diseases. A University College London team, which published the findings in Science Translational Medicine, featured a gene therapy encoding the dopamine transporter SLC6A3 for the treatment of a subtype of infantile parkinsonism caused by mutations in the gene. In mice, intraparenchymal stereotactic injection of an adeno-associated virus (AAV) vector encoding SLC6A3 extended survival and prevented the development of parkinsonism features without inducing the cortical cell loss and vacuolation seen with intracerebroventricular injection...