Section outline

  • Discussion Sessions: Cell, tissue and gene therapies
    History and regulatory framework for cell, tissue, and gene therapies. How these products are different from drugs and conventional biologics. Challenges for CMC development (donor sources, biologic variability, stability, assays), preclinical animal studies, and clinical trials. For gene therapies: gene transfer methods, special safety considerations for preclinical studies and clinical trials. Product examples including stem/progenitor cells, gene-modified cells, and cell/device combinations.

    Faculty:  Richard Chin

    Location:     China Basin 6702

    • Session Slides:

    • Epi 260 Read lecture 1 050912-1 File
    • Epi 260 Read lecture 2 050912 File

    • Session Audio/Video Recording (Access restricted to registered students):

    • Required Reading:

    • Carpenter MK, Frey-Vasconcells J, Rao MS. Developing safe therapies from human pluripotent stem cells File
      Not available unless: Your ID number contains 02

    • Optional Reading:

    • Dunbar CE, Larochelle A File
      Not available unless: Your ID number contains 02

    • Assignment:

      The assignment for gene therapy is to read the S-1 for Bluebird Bio - http://www.nasdaq.com/markets/ipos/filing.ashx?filingid=8882306  and comment on one or more of the following topics:

      1. How can Bluebird price its therapy?
      2. What are the issues Bluebird might face regarding patents
      3. Why did it take so long for Bluebird to get to where it is today?

       

    • Assignment Due Date:  May 28, 2014