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Overview
On-Demand Complimentary Webinar
Oligonucleotide-Based Therapeutics in Rare Diseases
Insights Guide to Oligonucleotide-Based Therapeutics
On-Demand
This webinar will examine how oligonucleotide therapeutics can be designed to modulate gene expression through multiple mechanisms and beneficially impact genetically defined rare diseases.
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This is a very targeted meeting and a "must attend" for those developing drugs based on oligonucleotides. It is a multidisciplinary meeting that is well organized and has several new and challenging topics where industry and regulatory agency attendees participate with a view to enabling getting drugs to patients.
-Previous Attendee
This program has been developed in collaboration with the DIA Oligonucleotide Scientific Working Group and is approved by the Regulatory Affairs Professionals Society for 12 RAC credits.
Who should attend?
Senior-level professionals and those working in the following areas of oligonucleotide science:
- Biotechnology
- Clinical Pharmacology
- Clinical Research
- Chemistry, Manufacturing, and Control
- Clinical, Regulatory, and Business Development
- Delivery Technologies
- Drug Discovery
- Preclinical
- Quality Assurance
- RNAi
- Vaccines
Learning objectives
At the conclusion of this Conference, participants should be able to:
- Identify accomplishments and challenges in the clinical development of oligonucleotide-based therapeutic drugs
- Describe the critical issues in the nonclinical development of oligonucleotides
- Differentiate the chemistry, manufacturing, and controls challenges associated with the development of synthetic oligonucleotides, including formulation and specification issues
- Explain unique aspects and various scientific approaches used during the development of oligonucleotide-based therapeutics
- Recognize the achievements made in the field to date and be able to share the vision with patients about the therapeutic potential that oligonucleotides possess across a wide range of indications
- Discuss industry and regulatory agency efforts to partner and address the unmet medical needs of patients
Program Committee
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James D. Thompson, PhD • Head, CMC Project Management
Moderna Therapeutics, United States -
James Wild, PhD • Pharmacologist, CDER
FDA, United States -
Paul C. Brown, PhD • ODE Associate Director for Pharmacology and Toxicology, OND, CDER
FDA, United States -
Daniel Capaldi, PhD • Vice President, Analytical and Process Development
Ionis Pharmaceuticals, Inc., United States -
Xuan Chi, MD, PhD • Pharmacologist/Toxicology Reviewer, Div. of Cardiovascular & Renal Products, OND
FDA, United States -
Robert T. Dorsam, PhD • Associate Director, Pharmacology/Toxicology, Office of Generic Drugs, CDER
FDA, United States -
Scott Henry, PhD • Vice President, Nonclinical Development
Ionis Pharmaceuticals, Inc., United States -
Aimee L. Jackson, PhD • Senior Director of Research
miRagen Therapeutics, United States -
Arthur M. Krieg, MD • Founder and CSO
Checkmate Pharmaceuticals, United States -
Arthur A. Levin, PhD • Executive Vice President, Research and Development
Avidity Biosciences , United States -
Jennifer Marlowe, PhD • Senior Director, Preclinical Development
bluebirdbio, United States -
Saraswathy V. Nochur • Senior Vice President, Regulatory Affairs & QA
Alnylam Pharmaceuticals, Inc., United States -
Emily J. Place, PhD, MPH • Pharmacologist, Office of New Drugs, CDER
FDA, United States -
Ramesh Raghavachari, PhD • Chief, Branch I, DPMA1, OLDP, OPQ, CDER
FDA, United States -
Kim Tyndall • Consultant
United States
