Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying treatment strategy.

As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.

Transferrin receptor (TfR)-mediated transcytosis is a receptor-mediated mechanism for drug delivery to the brain or other tissues, where, after the antibody binds to transferrin, the therapeutic agent is internalized and released into the brain.