Advances in oligonucleotide therapeutics: FDA guidance for pharma professionals

By Dr Nicola Davies

Oligonucleotide therapeutics are rapidly emerging as a promising class of drugs capable of addressing a variety of genetic and complex diseases by targeting RNA sequences.1

In June 2024, the US Food and Drug Administration (FDA) released a comprehensive guidance document titled Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics, providing a structured approach for pharmaceutical professionals working in this space.2 This article explores critical insights from the guidance and discusses how they shape the development, safety, and regulatory processes for oligonucleotide therapies.