{"id":22663,"date":"2024-11-29T13:16:24","date_gmt":"2024-11-29T12:16:24","guid":{"rendered":"https:\/\/kymos.com\/news\/nova-guia-de-la-fda-per-a-terapeutiques-amb-oligonucleotids\/"},"modified":"2025-08-06T20:31:49","modified_gmt":"2025-08-06T18:31:49","slug":"nova-guia-de-la-fda-per-a-terapeutiques-amb-oligonucleotids","status":"publish","type":"post","link":"https:\/\/kymos.com\/ca\/news\/nova-guia-de-la-fda-per-a-terapeutiques-amb-oligonucleotids\/","title":{"rendered":"Nova guia de la FDA per a terap\u00e8utiques amb oligonucle\u00f2tids"},"content":{"rendered":"

In June 2024, the FDA published the final version of the guideline titled \u201cClinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance for Industry\u201d. <\/strong>This document replaces the draft from 2022 and provides recommendations to assist the industry in the development of oligonucleotide therapeutics, but it also poses new challenges for developers and manufacturers. <\/span><\/p>\n

Previously, Maria Fauth, Nucleic Acids Department Manager at Kymos<\/strong>, shared her experience implementing the draft guideline back in 2022<\/a>, and now our team has revised this final version and prepared a summary of the most critical aspects and our experience on them.<\/span><\/p>\n

Oligonucleotide Therapeutics and the New Guideline<\/strong><\/h2>\n

Oligonucleotide therapies modulate gene expression to treat a specific condition or pathology by RNase H-mediated cleavage targeted degradation, splicing modulation, non-coding RNA inhibition, gene activation, and programmed gene editing (1). The development of Oligonucleotide Therapies has been arising in the latest years and these therapies and novel drugs include Antisense Oligonucleotides (ASO), siRNA, anti-miRs or antagomirs (anti-miRNA oligonucleotides), Aptamers, Clustered regularly interspaced palindromic repeats (CRISPR) and chemically modified oligonucleotides (1,2)<\/span><\/p>\n

Since early 2020, different oligonucleotide therapies have been approved by the FDA. Nonetheless, the broad use of these therapies has encountered some challenges, being the most significant ones: the achievement of an efficient delivery to target tissue besides the liver and having some interactions and toxicity (1,3).<\/span><\/p>\n

Luckily the FDA has recently released the final version of the Guideline for Oligonucleotide Therapeutics, <\/strong>highlighting the following considerations in the development of these new therapies:<\/span><\/p>\n

    \n
  1. Characterizing QTc Interval Prolongation and Proarrhythmic Potential (evaluated by the clinical site)<\/span><\/li>\n
  2. Performing Immunogenicity Risk Assessments<\/span><\/li>\n
  3. Characterizing the Impact of Hepatic and Renal Impairment on Pharmacokinetics, pharmacodynamics and Safety.<\/span><\/li>\n
  4. Considerations of Assessing Drug-Drug Interactions.<\/span><\/span><\/li>\n<\/ol>\n

    Kymos Group Experience with Nucleic Acids<\/strong><\/h2>\n

    Kymos Nucleic Acid Department has wide experience<\/strong> in the Bioanalysis of different Nucleic Acids and Oligonucleotide Therapies, even pioneering miRNA bioanalysis quantification since 2016<\/a>. Our department, led by Maria Fauth, <\/strong>has expertise in developing methods from scratch, and above all they have been implementing FDA guidelines in the GLP-compliant validation of our analytical methods since the draft release in 2022, always keeping track of any latest updates of the FDA.<\/span><\/p>\n

    Based on these current Guidelines Considerations and using state-of-the-art technology, Kymos is able to provide expertise in:<\/span><\/p>\n