ATH-1105, a small molecule positive modulator of the neurotrophic HGF system, demonstrates significant neuroprotective effects and extends survival in preclinical models of ALS. The study highlights the therapeutic potential of ATH-1105 in slowing or stopping neurodegeneration.
Neoleukin Therapeutics announces a novel protein decoy NL-CVX1 that blocks SARS-CoV-2 infection in vitro. The lead molecule is shown to protect hamsters from lethal doses of the virus through intranasal administration. This development demonstrates the potential of de novo protein design technology for addressing biological problems.
A new publication in Molecular Therapy outlines the dramatic effects of an exon skipping PPMO in preventing severe deterioration of dystrophin-deficient mice. The treatment restores dystrophin protein expression and maintains near-normal muscle function, suggesting great potential for treating Duchenne muscular dystrophy patients.