Capital Hilton

28 oct 2024 8:30 a.m. - 30 oct 2024 12:40 p.m.

1001 16th Street NW, Washington, DC 20036-5794

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 4 Track 1 and 2: Applying Toxicology Testing to the Clinic

Session Chair(s)

Elena  Braithwaite, PhD

Elena Braithwaite, PhD

Toxicologist

FDA, United States

Andrew  Slugg, MBA, MS

Andrew Slugg, MBA, MS

Senior Vice President, Global Head of Regulatory Affairs

Alnylam Pharmaceuticals, United States

Due to the unique pharmacokinetic properties of some oligonucleotide-based therapeutics, it can be challenging to compare the exposure achieved in animal pharmacology or toxicology studies to humans. Furthermore, understanding the relevant concentration of drug at the site of action (on and off target pharmacology and DDI) is key to the prediction of clinical outcomes. With a lack of clear guidance, several strategies have been employed to assess the relevance of nonclinical findings and predict clinical efficacy or safety. This session will share case examples of how programs have navigated from preclinical to clinical development including strategies used to calculate safety margins and DDI Liability with the goal of better predicting clinical outcomes.

Learning Objective : At the conclusion of this session, participants should be able to:
  • Define different parameters including pharmacokinetic metrics that can be used to calculate safety margins
  • Describe how in silico PK/PD models can be used in combination with cellular and subcellular kinetics to predict drug concentrations at the site of action

Speaker(s)

Steve  Hood, PhD

Refinement of PKPD Models and DDI Assessment

Steve Hood, PhD

GlaxoSmithKline, United Kingdom

Senior Director, Oligo ADME Strategy

Meena  Meena, PhD

Considerations for Determining Safety Margins for Oligonucleotides During Clinical Development

Meena Meena, PhD

Stoke Therapeutics, United States

SVP of Translational DMPK and Clinical Pharmacology

Julie  Douville, PhD, MS

Harnessing ASO Platform Technology: A Precise Approach to the Design, Development and Administration of Individualized Therapies

Julie Douville, PhD, MS

N-Lorem Foundation, United States

Executive Director, ASO Discovery and Development

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