The FDA paper titled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics” was released in June 2022. The paper provides guidance on the development of oligonucleotide therapeutics, which are a promising new class of drugs that target genetic material.
The paper provides a comprehensive overview of the pharmacology of oligonucleotide therapeutics, including their mechanism of action, pharmacokinetics, pharmacodynamics, and toxicity. It also discusses the regulatory requirements for the development and approval of these drugs.
In the context of this last point, the regulatory requirements could be a limiting factor for the development of generic oligonucleotides, especially regarding the need to show control of the diastereomeric sameness for each step of the process.
Brick42, together with research partners in Switzerland and Italy, is engaged in a study of a novel analytical approach to meet this challenge.