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Bethesda North Marriott Hotel and Conference Center

Apr 25, 2022 7:00 AM - Apr 27, 2022 12:00 PM

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

DIA/FDA Oligonucleotide-Based Therapeutics Conference

DIA Your Way! Join us live in-person or live from the comfort of your home/office. This event offers two learning avenues depending on your preference!

Session 8 Track 1 and 2 – Individualized Antisense Oligonucleotide Drug Products

Session Chair(s)

Timothy W. Yu, MD, PhD

Timothy W. Yu, MD, PhD

Associate Professor, Harvard Medical School; Division of Genetics and Genomics

Boston Children’s Hospital, United States

Ronald  Wange, PhD

Ronald Wange, PhD

Associate Director for Pharm/Tox, OND, CDER

FDA, United States

The availability of annotated, complete human genome databases, and the ability to conduct whole-genome sequencing rapidly and cheaply, have given individual clinicians tools that can allow them to pinpoint the exact genetic cause of a given patient’s disease. This makes it possible for clinicians to rapidly identify experimental oligonucleotide-based drug candidates that are tailored to an individual patient’s specific disease-causing genetic variant. To support this important advance in treatment availability for those with very rare genetic diseases, the US FDA has recently published a series of draft guidances to aid sponsor investigators in developing these “n of 1” treatments. This session will include presentations on the recommendations contained in the draft guidances as well as examples of the great potential and challenges associated with developing these products.

Learning Objective :
  • Describe the nonclinical information that FDA recommends to support an investigational new drug application (IND)
  • Recognize key molecular genetic and clinical criteria by which candidates for investigational “n of 1” intervention are being selected
  • Understand the evolving landscape of stakeholders in the US and abroad invested in studying the safety, efficacy, and scalability of “n of 1” treatments, and the key opportunities and challenges they are addressing


Peter P. Stein, MD

Regulatory Considerations in Developing Individualized ASOs: Challenges and Approaches: Peter P. Stein, MD

Director, Office of New Drugs, CDER

FDA, United States

Timothy W. Yu, MD, PhD

Presentation: Timothy W. Yu, MD, PhD

Associate Professor, Harvard Medical School; Division of Genetics and Genomics

Boston Children’s Hospital, United States

Annemieke Aartsma-Rus Aartsma-Rus, PhD, MSc

N of 1 Development: The Global Perspective: Annemieke Aartsma-Rus Aartsma-Rus, PhD, MSc

Professor, Translational Genetics

LUMC, Netherlands

Sarah  Glass, PhD

Panelist: Sarah Glass, PhD

Chief Operating Officer

N-Lorem Foundation, United States

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