The N-of-1+ initiative is focused on the development of antisense oligonucleotide (ASO) therapeutics for patients with a serious, life-threatening rare disease where there are fewer than ~100 known patients worldwide.
The rapid progress in drug development for n=1 and ultra-rare diseases raises multiple complex issues. The OTS Rare Disease Task Force was created to help address some of these issues.
As the main non-profit society in this field, we seek to help in three ways:
- Formulating guidelines to help avoid wasted effort and dangerous projects
- Helping to streamline the process of drug development and approval by connecting patients/families/physicians with appropriate resources
- Helping to clarify expectations, guidelines, and protocols ideally in dialogue with the FDA