Preclinical research of iPS Cell Therapy

iPS Cells and Therapeutic Applications for Duchenne

Questions and Answers

What stage is this research?

We are currently in the optimization/validation phase of preclinical development.

Where is this research being done and who is funding this research?

This research is being done in the lab of Dr. Rita Perlingeiro at the University of Minnesota, in partnership with the University of Minnesota Center for Translational Medicine and the Molecular and Cellular Therapeutics Facility. This work is currently funded by the Department of Defense (DoD).

What is the goal or purpose of this research?

Induced pluripotent stem cells (iPS) are adult cells that have been reprogrammed to an embryonic stem cell-like state.
There has been tremendous excitement for the therapeutic potential of iPS cells in treating genetic diseases. Our current research builds on our successful proof-of-principle studies for Duchenne performed with mouse wild-type and dystrophic iPS cells as well as control (healthy) human iPS cells. These studies demonstrate equivalent functional myogenic engraftment to that observed with their embryonic counterparts following their transplantation into dystrophic mice.

Our goal now is to apply this technology to clinical grade GMP-compliant iPS cells, and generate a cell product, iPS-derived myogenic progenitors, that can be delivered to muscular dystrophy patients.

What steps need to be completed before moving into a clinical trial?

Optimization of methodology, characterization of cell product, scalability with GMP-compliant method, followed by safety and efficacy studies. Once these have been achieved, we will be ready to move into a clinical trial.

What is your best estimate for the length of time it will take to move this research into clinical trials?

3-4 years (it depends largely on how much funding we have available to conduct these studies).

Where would a clinical trial take place?

University of Minnesota

Who would be eligible to participate in a clinical trial?

To be defined

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