Considerations for Oligonucleotide Therapeutics in CMC

Guidance in navigating through the regulatory grey area

Oligonucleotide therapeutics are an emerging field with over a dozen new oligonucleotide-based drugs approved globally since 2016. The well-established chemical process for synthesizing oligonucleotides gives access to ‘large small’ molecules of sizes between 5000 and 20,000 Daltons and positions oligonucleotide therapeutics between small molecules and biologicals.

Assessing the quality and specifications is often a challenge in this regulatory grey area because the International Council for Harmonisation (ICH) guidelines consider this modality as a chemical entity while sharing properties with biologics.

Therefore, a thorough understanding of oligonucleotide’s mechanism of action, safety aspects and the production process helps with informed decision-making for the chemistry, manufacturing and controls’ (CMC) critical quality attributes (CQAs).

In this webinar, the expert speakers will share insights on oligonucleotide therapeutics as an emerging therapeutic modality and focus on their production processes, drug substance specifications, material properties and potential impurities. They will also explore CMC Investigational Medicinal Product (IMP) considerations specific to oligonucleotides, including the identification of CQAs and critical process parameters (CPPs) essential for drug development.

They will also discuss key manufacturing aspects, as well as stability and scalability concerns for oligonucleotide therapeutics, to ensure a thorough understanding of the production lifecycle. Moreover, this webinar will provide guidance on progressing into early-phase clinical studies and emphasize on how to set and meet specifications to maintain quality and comply with regulatory standards, thus ensuring a smooth transition from development to clinical evaluation.

Register for this webinar today to gain expert insights into the complex regulatory landscape of oligonucleotide therapeutics.

Speakers

André Boltjes, PhD, Project Director CMC, ICON

André Boltjes is a Project Director CMC at ICON. He is involved in technical discussions related to CMC strategies and supports clients in formulating their API into a fit-for-purpose drug product to support their clinical studies. André provides guidance in method and formulation development, stability studies and various study designs, including first-in-human and ADME/BA studies (radiosynthesis).

Message Presenter

Pascal van Tilburg, BSc, Senior Project Manager and SME in CMC, ICON

Pascal van Tilburg is a Senior Project Manager and Subject Matter Expert (SME) in CMC. With almost three decades of experience in QC and general IMP development programs, he has seen the overall GMP manufacturing field develop into its current form. This invaluable experience provides expert guidance through the ever-changing landscape of CMC.

Message Presenter

Jasper Boonstra, PharmD, PhD, Qualified Person, ICON

Jasper Boonstra is a Qualified Person at ICON. He is involved in quality discussions related to formulations to be developed and administered to subjects in early-phase trials primarily first-in-human and ADME/BA studies. Jasper provides input on quality-related challenges prior and during the clinical program.

Message Presenter