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Session 8 Track 1 and 2 – Individualized Antisense Oligonucleotide Drug Products
Session Chair(s)
Timothy W. Yu, MD, PhD
Associate Professor, Harvard Medical School; Division of Genetics and Genomics
Boston Children’s Hospital, United States
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
Speaker(s)
Regulatory Considerations in Developing Individualized ASOs: Challenges and Approaches
Peter P. Stein, MD
FDA, United States
Director, Office of New Drugs, CDER
Presentation
Timothy W. Yu, MD, PhD
Boston Children’s Hospital, United States
Associate Professor, Harvard Medical School; Division of Genetics and Genomics
N of 1 Development: The Global Perspective
Annemieke Aartsma-Rus Aartsma-Rus, PhD, MSc
LUMC, Netherlands
Professor, Translational Genetics
Panelist
Sarah Glass, PhD
N-Lorem Foundation, United States
Chief Operating Officer
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