DIA/FDA Oligonucleotide-Based Therapeutics Conference

CRISPR-Cas gene editing is unique among all genetic therapy modalities in relying on an RNA oligonucleotide to target a number of clinical-need-determined molecular genetic outcomes to any native gene. Definitive clinical evidence illustrates the curative potential of gene editing in the hemoglobinopathies, TTR amyloidosis, and hereditary angioedema. Further progress of this oligonucleotide-based therapeutic modality is hampered by lack of a platform approach to the design, development, and delivery of an on-demand, patient-specific gene editor at a time and a cost commensurate with benefit-risk considerations. Robust efforts to address these challenges are underway, with clinical trial evidence from the deployment of such platform approaches being critical to essential streamlining of the nonclinical and CMC path.