Overview:
Oligonucleotide therapeutics are an emerging field with over a dozen new oligonucleotide drugs approved globally since 2016. This well-established chemical process for synthesizing oligonucleotides gives access to ‘large small’ molecules between 5000 to 20,000 daltons and positions this therapeutic modality between small molecules and biologicals.
Assessing the quality and specifications is often a challenge in this regulatory grey area, given the fact that the ICH (International Council for Harmonisation) guidelines consider this modality as a chemical entity while sharing properties with biologics
A thorough understanding of the molecule’s mechanism of action, safety aspects and the production process therefore helps with informed decision making for the CMC critical quality attributes.
During this webinar, we will discuss::
1) Oligonucleotides - a young therapeutic modality
- Production process and drug substance specifications
- Material properties and impurities
2) CMC IMP considerations for Oligonucleotides
- Understanding the critical quality attributes (CQAs) and critical process parameters (CPPs) in oligonucleotide drug product development
- Key aspects of manufacturing, stability, and scalability for oligonucleotide drug products
3) Progression into early phase studies
- Guidance on setting and meeting specifications to ensure quality and compliance
Speakers:
André Boltjes, PhD
André 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).
Pascal van Tilburg, BSc
Pascal 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.
Jasper Boonstra, PharmD, PhD
Jasper 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.