DIA/FDA Oligonucleotide-Based Therapeutics Conference

Convening industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development.

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Session 7A: Recently Approved and Late-Stage Oligonucleotide Drugs

Session Chair(s)

G. Susan Srivatsa, PhD

President

ElixinPharma, United States

This session will cover recent experience with approved and late-stage oligonucleotide drugs. The first presentation will address the CMC challenges associated with the review and approval of Nusinersen, and the second will cover regulatory experience with late-stage development of an siRNA drug. There will be a panel discussion that may include representatives from the FDA and BfArM.

Learning Objective :
Understand CMC requirements for late-stage and development market approval of oligonucleotides in the US and other regions
Identify CMC areas that may be important for late stage development of therapeutic oligonucleotides

Speaker(s)

SPINRAZA (nusinersen) Approval: CMC Strategies and Lessons Learned

Firoz Antia, PhD

Denali Therapeutics, United States

Vice President, Oligonucleotide and Small Molecule CMC

CMC Strategies for Late-Stage Development of siRNA Oligonucleotides

Vidhya Gopalakrishnan, PhD

Quark Pharmaceuticals, Inc, United States

Senior Vice President, Pharmaceutical Development

Panel Discussion

All Session Speakers, United States

Panelist

Olen M. Stephens, PhD

FDA, United States

Chemist Reviewer, CMC Reviewer, CDER/OPQ/ONDP

Panelist

Daniel Capaldi, PhD

Ionis Pharmaceuticals, Inc, United States

Vice President, Analytical and Process Development

DIA/FDA Oligonucleotide-Based Therapeutics Conference

Session 7A: Recently Approved and Late-Stage Oligonucleotide Drugs

Session Chair(s)

G. Susan Srivatsa, PhD

Learning Objective : Understand CMC requirements for late-stage and development market approval of oligonucleotides in the US and other regions Identify CMC areas that may be important for late stage development of therapeutic oligonucleotides

Speaker(s)

SPINRAZA (nusinersen) Approval: CMC Strategies and Lessons Learned

CMC Strategies for Late-Stage Development of siRNA Oligonucleotides

Panel Discussion

Panelist

Panelist

获得信息并保持参与

Learning Objective :
Understand CMC requirements for late-stage and development market approval of oligonucleotides in the US and other regions
Identify CMC areas that may be important for late stage development of therapeutic oligonucleotides