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Translational Clinical Pharmacology
Symposia

Symposium 17: Hot Topics: Advancements in Antisense Oligonucleotide Development: Challenges, Strategies & Novel Approaches

Tuesday, September 10, 2024
3:30 PM – 5:00 PM ET
Location:Grand Ballroom D
CE: 1.5

Target Audience:

The target audience for this session includes PharmD, MD and PhD scientists from industry, academia and regulatory agencies interested in antisense oligonucleotide (ASO) development, as well as clinicians working with ASO treatment clinically.



Description:

Antisense oligonucleotides are an emerging and rapidly growing modality that has the potential to revolutionize the treatment of diseases with a high unmet need in multiple therapeutic areas. The identification of new molecular targets combined with the targeted approach of ASOs creates the opportunity to develop treatments for rare genetic disorders that have thus far been untreatable with available therapeutic approaches. For example, intrathecally administered ASOs have proven clinical effect in the treatment of a variety of neuromuscular disorders caused by genetic abnormalities or increased protein accumulation. The versatility of ASOs also enables its application to more common diseases with multifactorial etiologies. ASOs are designed to recognize RNA and activate or down-regulate a specific component of the pathway of the disease. Compared to other modalities, ASOs are unique in several ways, including the mechanism of action, structure, chemical modifications to the base, size, sequence, delivery strategy and any other moieties such as small molecules, proteins or antibodies. Due to the novel nature of this modality, identifying the ideal dose regimen, appropriate delivery and measuring efficacy can be uniquely challenging. Additionally, due to the distinct characteristics of ASOs, special considerations may need to be taken into account when evaluating cardiac safety, immunogenicity risk, impacts of organ impairment and drug-drug interactions. This Symposium will elucidate the key clinical pharmacology, pharmacometrics and regulatory strategies for advancing ASO drug development and integration into clinical applications. Distribution of ASOs after different routes of administration including systemic and intrathecal delivery will be discussed.

Goals & Objectives:

Following the completion of this activity, the learner will be able to:
  • Describe basic principles that underline ASOs and the current status of the market;
  • Explain mechanisms of ASO distribution and pharmacology across different therapeutic areas and methods of administration;
  • Discuss the unique regulatory considerations and challenges associated with ASOs;
  • Identify cases in which model-informed drug development could be used to optimize ASO development.

Presentations:

  • Decoding the ASO Landscape: An Overview

    Co-Chair & Faculty: Reena Kartha, MS, PhD (she/her/hers) – Ctr for Orphan Drug Research, Coll of Pharmacy, Univ of Minnesota

  • Overcoming Obstacles & Pioneering Progress: Clinical Pharmacology Considerations for ASO Development

    Co-Chair & Faculty: Natalie Schmitz, PharmD, PhD – Biogen Inc

  • ASO Neurological Therapy: Exploring Intrathecal Administration via Physiologically-based Pharmacokinetic Modeling

    Faculty: Michael Monine, PhD – Biogen Inc

  • Navigating the Regulatory Pathway of ASO Therapeutics

    Faculty: Emily Noonan Place, PhD, MPH (she/her/hers) – Aclairo Pharmaceutical Development Group Inc

  • Discussion, Questions & Answers, Learner Feedback & Evaluations