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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.
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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
-
James D. Thompson, PhD • CMC Therapeutic Area Lead
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 Capaldi, PhD • Vice President, Analytical and Process Development
Ionis Pharmaceuticals, Inc, United States -
Xuan Chi, MD, PhD • Supervisory Pharmacologist
CDER, 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 • Chief Scientific Officer
Atalanta Therapeutics, United States -
Arthur M. Krieg, MD • CEO and CSO
Checkmate Pharmaceuticals, United States -
Arthur A. Levin, PhD • CSO
Avidity Biosciences , United States -
Jennifer Marlowe, PhD • Senior Director, Preclinical Development
bluebirdbio, United States -
Saraswathy V. Nochur, PhD, MSc • Chief Regulatory Officer
Alnylam Pharmaceuticals, United States -
Emily Place, PhD, MPH • Senior Consultant
Biologics Consulting, United States -
Ramesh Raghavachari, PhD • Chief, Branch I, DPMA1, OLDP, OPQ, CDER
FDA, United States -
Kim Tyndall • President
CMC Tyndall Consultant LLC, United States
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On-Demand Complimentary Webinar
Oligonucleotide-Based Therapeutics in Rare Diseases
