A US Food and Drug Administration draft guidance on clinical considerations for individualized antisense oligonucleotide drug products emphasizes the limited target population for such treatments.
Antisense Oligonucleotide Clinical Guidance Emphasizes Individualized Nature Of Treatment
US FDA’ s latest ‘n of 1’ guidance describes clinical considerations for INDs to support dosing and monitoring of an individual with a life-threatening genetic disease who is eligible to receive an ASO; recommendations apply only when a few patients may be candidates for treatment.
