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# Antisense Oligonucleotides Guidance Analyzed
- URL: https://www.fdaweb.com/antisense-oligonucleotides-guidance-analyzed/
- Published: 2021-02-04T12:00:00.000Z
- Updated: 2026-09-14T16:45:31.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5148595

Three Morgan Lewis attorneys say a recent FDA [guidance](http://www.fda.gov/media/144872/download?ref=fdaweb.com) to facilitate the development of antisense oligonucleotide (ASO) drugs for patients with severely debilitating or life-threatening genetic disorders that are tailored to a patient’s specific genetic variant is targeted to academic investigators who may be less familiar with the agency’s requirements and less experienced in interacting with FDA. In an online [analysis](https://www.morganlewis.com/pubs/2021/02/fda-issues-more-guidance-on-gene-and-cell-therapy-products-cv19-lf?ref=fdaweb.com), the attorneys say that healthcare institutions where ASO products are used should familiarize themselves with FDA’s requirements and processes to ensure that any use of an investigational ASO product meets regulatory requirements.

“It will also be important that manufacturers supporting the use of ASO products or that later intend to work with ASO product investigators ensure that programs comply with FDA’s regulations via contractual agreement and, as appropriate, due diligence,” they add.

The post covers FDA guidance recommendations on early FDA interactions, confidentiality, submissions, and ethics.

The authors say the guidance is likely a first step in the development of individualized therapies. They quote FDA optimism that “development of \[ASO\] individualized drug products may spur gene sequencing that leads to the development of additional individualized drug products.” They say that through the guidance, the agency “aims to determine the most effective and efficient way to bring personalized drugs to patients while ensuring the right risk/benefit balance.”