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# Oligonucleotide-Based Therapeutic Assessment
- URL: https://www.fdaweb.com/oligonucleotide-based-therapeutic-assessment/
- Published: 2024-11-13T12:00:00.000Z
- Updated: 2026-09-14T14:46:58.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5158179

FDA has published a draft guidance, *Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics*, with recommendations on the approaches for the nonclinical safety evaluation of oligonucleotide-based therapeutics (ONTs) to support clinical development and marketing of those products. The document says although current guidance for small molecule drugs and therapeutic proteins may mention ONTs, this guidance provides detailed recommendations specific to the nonclinical assessment of ONTs that have been developed based on experience to date with this category of products. The recommendations address ONT characteristics that differ from small molecule drugs and therapeutic proteins, it says.

The guidance includes recommendations for single-stranded or double-stranded ONTs created synthetically or derived naturally, with native or modified backbone or nucleotide structures that increase or decrease the expression and/or function of proteins.

The guidance includes an Introduction, Scope, General Considerations, Pharmacology, Pharmacokinetics, General Toxicity Studies, Genotoxicity Studies, Reproductive and Developmental Toxicity Studies, Carcinogenicity Studies, Local Tolerance, Immunotoxicity, Photosafety, Other Toxicity Studies, and Notes.