This course is focused on the practical application of the principles of translating stem-cell-based therapies that focuses on the discovery, non-clinical development and Phase 1 clinical trials required to translate these interventions from the bench to the bedside. This course will highlight the unique aspects of stem-cell- based therapy discovery and development compared to drug discovery and development. Lectures given by a multidisciplinary team of academic subject matter experts will teach concepts that are linked to application of translational science principles to the discovery and development stem-cell-based therapeutics.
By the end of this course, the student should be able to:
Understand the bench to bedside concept
Understand the process of development of new stem-cell- based therapies
Understand the preclinical work needed to support first-in-human studies
Understand the discovery process in cell and gene therapy.
Describe the processes to deliver first-in-humans studies.
Understand basics for phase 1 first-in-human studies
Understand the regulatory landscape for cell and gene therapy.
Design a target product profile (TPP) for a cell or gene therapy product
Understand the human subject ethical issues
Course Number: CLRE-237
Credit: 2.00 unit(s)