This month in The Journal

Existing best practices for pre-clinical and clinical research are not well-suited for individualized therapeutic approaches that leverage new treatment modalities, such as antisense oligonucleotides (ASOs). The increasing number of individualized ASOs in clinical use has driven rapid changes in FDA regulatory frameworks, patient eligibility criteria, and approaches to clinical risk management. In a typical clinical trial setting, detailed consent occurs once, and extensive pre-clinical data provides an estimate of benefits and risks.