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

Available On-Demand
Defining A Regulatory Path For Induced Pluripotent Stem Cells Based Cell Therapy


Induced pluripotent stem (iPS) cells are a potential source for developing autologous cell therapies. Here we provide 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. We focus on age-related macular degeneration (AMD), a blinding eye disease that affects more than 30 million individuals worldwide. AMD is caused by the atrophy of retinal pigment epithelium (RPE), a monolayer neuroectodermal tissue that is located in the back of the eye. In our process, a biodegradable scaffold supports the RPE monolayer tissue during transplantation in the back of the eye. We have developed a clinically compatible GMP manufacturing process to generate and functionally validate AMD patient-specific iPSC-derived RPE monolayer tissue. The entire manufacturing process, from a patient’s blood cells to transplantable tissue, takes approximately 157 days. We have developed in-process quality controls to monitor the process and steps to validate patient-specific iPS cell banks. AMD patient-specific iPSC-derived RPE tissue is similar to the native RPE in its structural, molecular, and functional features. This human RPE tissue is able to rescue RPE damage-induced vision loss in two different animal models: a genetic retinal degeneration rat model and an RPE injury based pig model. We have developed surgical procedures and tools for transplanting this tissue into patients. Our work provides a streamlined regulatory path to develop cell therapies for iPS cells and a potential treatment for AMD.

Topics Covered Include:
  • Streamlining a path to file Investigational New Drug (IND) applications
  • 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


Kapil Bharti, Ph.D.

Kapil Bharti, Ph.D.

National Eye Institute
Unit on Ocular Stem Cells and Tranlational Research

Dr. Bharti holds a bachelor’s degree in biophysics from the Panjab University in Chandigarh, India, where he graduated with highest honors. This was followed by a master degree in biotechnology at the Maharaja Sayaji Rao University in Baroda, India and a diploma in molecular cell biology at the Johann Wolfgang Goethe University at Frankfurt in Germany. Supported by an international Ph.D. student fellowship, he obtained his Ph.D. from the same institution, graduating summa cum laude. His Ph.D. work involved basic biology in the areas of heat stress, cellular chaperones, and epigenetics. From Germany, Dr. Bharti came to the National Institute of Neurological Disorders and Stroke to work with Dr. Heinz Arnheiter as a postdoctoral fellow. While there, he published numerous papers in the areas of transcription factor regulation, pigment cell biology, and the developmental biology of the eye. It is perhaps this combination of diverse backgrounds that led him to develop an interest in the emerging field of stem cell biology, particularly of the retinal pigment epithelium, as he moved into the role of staff scientist. Dr. Bharti has authored numerous publications and has won several awards, including, most recently, being named an Earl Stadtman Investigator.