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.

Flowers in field
In FDA's view, a study for an individualized ASO therapy will involve 1 or 2 patients. • Source: Alamy

A US Food and Drug Administration draft guidance on clinical considerations for individualized antisense oligonucleotide drug products emphasizes the limited target population for such treatments.

The guidance describes clinical considerations for investigational new drug application (IND) submissions to support initial and continued administration, dosing and clinical monitoring of an individual with a severely debilitating and life-threatening genetic disease who is

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