Individualized antisense oligonucleotide (ASO) technology is increasingly capturing the attention of researchers, patients, clinicians, and regulators, among others, following the development of several promising ASO interventions for rare, devastating diseases. Yet the development of these therapies exists uncomfortably in our current regulatory and ethical frameworks, in which research and medical practice are separate endeavors. Likewise, current market-driven drug development practices cannot currently support devoting resources to individualized therapeutics, which will never be indicated for more than a handful of patients.
1. Gain familiarity with the ethical challenges facing the emerging field of individualized anti-sense oglionucleotide (ASO) therapeutics
2. Understand the overlapping roles of treatment and research in individualized therapeutics
3. Identify ways in which individualized therapies require unique regulatory and development consideration