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Bethesda North Marriott Hotel and Conference Center

Oct 28, 2019 7:00 AM - Oct 30, 2019 12:30 PM

5701 Marinelli Road, , North Bethesda, MD 20852 , USA

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 4 Track A & B: Evaluation of QT Prolongation Potential and Assessment of Immunogenicity

Session Chair(s)

Hobart  Rogers, PharmD, PhD

Hobart Rogers, PharmD, PhD

Pharmacologist, CDER, FDA, United States

Scott  Henry, PhD

Scott Henry, PhD

Senior Vice President, Nonclinical Development, Ionis Pharmaceuticals, Inc., United States

Oligonucleotide therapeutics have unique characteristics that often require differential pre-clinical and clinical assessment compared to small molecules. This session will address two of these topics: Topic One will focus on the nonclinical and clinical aspects of the assessment of QT prolongation potential; Topic Two will focus on the evaluation of the nonclinical and clinical aspects regarding immunogenicity. Both topics will have perspective presentations from both industry and regulatory speakers. Following the presentations, a joint panel will convene to further discuss these topics with implications for guiding future regulatory oversight.

Speaker(s)

Wendy  Wu, PhD

Wendy Wu, PhD

Pharmacologist, FDA, United States

Are Oligonucleotide Therapeutics Too “Large” to Interact with Cardiac Ion Channels: Insights from the FDA Database

James  Kramer, PhD

James Kramer, PhD

Principal Scientist, Charles River Laboratories, United States

Oligonucleotide Cardiovascular Safety Testing

Nan  Zheng, PhD

Nan Zheng, PhD

Scientific Lead, Office of Clinical Pharmacology/CDER, FDA, United States

Clinical QT Assessment for Oligonucleotide-Based Therapeutics: Regulatory Experience and Current Thinking

Yanfeng  Wang, PhD

Yanfeng Wang, PhD

Executive Director, Ionis Pharmaceuticals, United States

QT Evaluation for Antisense Oligonucleotides: Ionis Experience

  Panel Discussion

Panel Discussion

, All Session Speakers, United States

Panel Discussion

Zhenzhen  Liu

Zhenzhen Liu

Biologist, CDER, FDA, United States

FDA Presentation: Assessment of Immunogenicity Studies

Zhenrong  Yu, PhD

Zhenrong Yu, PhD

Executive Director, Ionis Pharmaceuticals, Inc, United States

Immunogenicity Assessments for Antisense Oligonucleotides

Daniel  Mytych, PhD

Daniel Mytych, PhD

Scientific Director, Clinical Immunology, Department of Translational Medicine, Amgen, Inc. , United States

Perspectives on the Designation of Oligonucleotide Starting Materials

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