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# Antisense Oligonucleotide Nonclinical Testing Guidance
- URL: https://www.fdaweb.com/antisense-oligonucleotide-nonclinical-testing-guidance/
- Published: 2021-04-27T12:00:00.000Z
- Updated: 2026-09-14T16:56:36.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5149195

FDA has published a draft guidance, *Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases*, to inform sponsor-investigators about the nonclinical information that the agency recommends to support an IND for an antisense oligonucleotide being developed to treat a severely debilitating or life-threatening disease caused by a unique genetic variant where only a small number of individuals are prospectively identified. The [document](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/nonclinical-testing-individualized-antisense-oligonucleotide-drug-products-severely-debilitating-or?ref=fdaweb.com) says the investigational antisense oligonucleotide should be from a well-characterized chemical class for which there is substantial nonclinical information and clinical experience that is publicly available or to which the sponsor-investigator has a right of reference.

The guidance is not intended to address nonclinical testing for the commercial development of oligonucleotides.

Contents of the guidance are Introduction, Proof of Concept, IND-supporting Safety Studies, FIH Dose Selection, Dose Escalation, and Factors Supporting Abbreviated Nonclinical Assessment Approach Described in This Guidance.