Scalable Manufacturing and Toxicology Infrastructure

to support N-of-1 trials, where:

• a small number of individuals are prospectively identified (typically one or two);
• no FDA-approved treatment options are/will be available;
• diseases are rapidly progressing, resulting in irreversible morbidity or early death.

Latest Posts:

• Designing Principles for Individualized ASOs
• Modifications permitted for individualized ASOs