Defining A Regulatory Path For Induced Pluripotent Stem Cells Based Cell Therapy

Friday, August 26, 2016 - 09:12

Kapil Bharti, PhD, National Eye Institute, Unit on Ocular Stem Cells and Translational Research

Induced pluripotent stem (iPS) cells are a potential source for developing autologous cell therapies. In this webinar, Dr. Kapil Bharti, provides a streamlined regulatory (cell manufacturing and preclinical) path towards filing an Investigational New Drug (IND) application to conduct a phase I clinical trial using autologous iPS cells. His group has developed a clinically compatible Good Manufacturing Practices (GMP) process to generate and functionally validate age-related macular degeneration (AMD) patient-specific iPS cell-derived retinal pigment monolayer tissue. He will discuss in-process quality controls that they have developed to monitor the entire manufacturing process and steps to validate patient-specific iPS cell banks. This human RPE tissue his group has developed using these processes is able to rescue RPE damage induced vision loss in two different animal models and provides a streamlined regulatory path to develop cell therapies for iPS cells and a potential therapy for AMD.

Topics covered include:

Streamline path to file and Investigational New Drug (IND) application
Conducting a phase I clinical trial using autologous induced pluripotent stem cells
Developing a clinically compatible GMP manufacturing process
Quality controls to monitor the entire manufacturing process
Generating and functionally validating patient-specific iPSC-derived retinal pigment epithelium monolayer tissue.