Biopharmaceuticals (includes Biologics)

Strategic

Advanced Therapies and RNA-based Therapeutics: Opportunities and Challenges

Session Leader(s)

• 

  Kevin Healy, PhD, RAC

  Vice President, Regulatory Affairs and Quality
  Enzyvant Therapeutics

Speaker(s)

• 

  Sabine Haubenreisser, MSc, PhD

  Principal Scientific Administrator
  European Medicines Agency
• 

  Tracy Meffen, MS, CQA, RAC

  Vice President, Quality & Regulatory Affairs
  Genevant

Advanced therapies, including cell and gene therapies, tissue engineered products, and cell and tissue products, are being developed throughout the biotechnology and pharmaceutical industry to treat serious and life-threatening diseases. The development of advanced therapies and nucleic acid drugs is challenging, but global health authorities have established regulatory designations and pathways to help expedite development. In this session, the speakers will provide an overview of the regulatory challenges and opportunities, and discuss their experiences developing a tissue engineered product and RNA-based therapeutics. The session will highlight some of the unique characteristics of developing mRNA and RNAi therapeutics, including discussion of indication selection and their fit within US and European regulatory frameworks.

 

Learning Objectives:

• Develop an understanding of the requirements and benefits associated with at least 3 different FDA or EMA designations for the expedited development and approval Advanced Therapies.
• Describe two types of nucleic acid drug and the major regulatory considerations in the US and Europe.
• Explain the unique CMC characteristics of mRNA and RNAi drugs.