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Stem Cell Technology Update: The Rise of Induced Pluripotent Stem Cells

Decision Resources, Inc, March 2010, Pages: 21

Scientists achieved a significant breakthrough in stem cell research with the development of induced pluripotent stem (iPS) cells—adult cells that are “reprogrammed” to be pluripotent. Many scientists believe that stem cells are the key to successful therapies for incurable diseases and conditions such as type 1 diabetes, spinal cord injuries, and neurodegenerative diseases such as Parkinson’s disease and ALS. However, research on stem cells over the past decade has been stymied in part by ethical concerns and governmental policies that restrict or ban research on embryonic and fetal tissue. iPS cell technology—although still in its infancy—offers a clear path around many of the ethical issues associated with embryonic stem cells and introduces the potential for substantial strides in this area of research. In fact, in January 2010, Nature Methods named “induced pluripotency as a tool for biological discover” as its Method of the Year 2009—just one reflection of this technology’s importance.

Questions Answered in this Report:

- iPS cells offer scientists and drug companies the opportunity to utilize the pluripotency of stem cells without having to work with human embryos. What is the current state of iPS cell research? What are the technology’s strengths and weaknesses?

- iPS cell technology is in its early stages, and therapeutic applications of this technology are still many years away. What advancements need to occur for iPS cells to become a viable therapeutic technology? What are the near-term opportunities for iPS cell technology?

- In February 2010, Fate Therapeutics was awarded a U.S. patent for the reprogramming of somatic cells that the company has heralded as the fi rst patent awarded for iPS technology. What work is ongoing at other start-up companies focusing on the use of iPS technology? Which companies are leading the commercialization of iPS cell technology? How are Big Pharma and drug discovery companies incorporating iPS cells into their platforms?

Scope:

- Technologies covered: Human embryonic stem cells (hES), somatic cell nuclear transfer (SCNT), induced pluripotent stem cells (iPS).

- Opportunities for iPS research: Cellular models, approaches to factor-free iPS cells, iPS cell models that mimic in vivo environments.

- Profiles in innovation: Harvard Stem Cell Institute, Fate Therapeutics, Cellular Dynamics International, iPierian.

- Current and future disease targets of stem cell research: Amyotrophic lateral sclerosis, spinal muscular atrophy, familial dysautonomia, type 1 diabetes, Parkinson’s disease.
- Outlook for iPS cell technology: The therapeutic potential of iPS cells, Big Pharma’s interest in
iPS technology.

Key Terms for This Report:

Embryonic stem cells, Induced pluripotent stem cells (iPS), Somatic cell nuclear transfer (SCNT), Stem cell bans, Stem cell research, Stem cell therapy, Parkinson’s disease, Cloning, Amyotrophic lateral sclerosis (ALS), Animal models, Drug discovery, Neurodegenerative disease, Type 1 diabetes, Fetal tissue research, Spinal cord injury

Executive Summary
Strategic Considerations
Stakeholder Implications
Introduction

An Overview of Stem Cells
Human Embryonic Stem Cells
Somatic Cell Nuclear Transfer
Human Induced Pluripotent Stem Cell Technology

Near-Term Applications for iPS Technology
Designing “Disease in a Dish” Models
Challenges to the Development of Human iPS-Cell-Based Disease Models
Factor-Free iPS Cells
Creating In Vitro Disease Models That Resemble In Vivo Environments

Profiles in Innovation
Harvard Stem Cell Institute
Fate Therapeutics
iPierian
Cellular Dynamics International

Outlook

Tables
1. Stem Cell Policies in the Major Markets
2. Select Companies Developing hES Cells as Therapeutics
3. Select Diseases with iPS Cellular Models
4. Key Opportunities and Challenges or iPS Technology

Figures
1. Timeline of U.S. Stem Cell Policy
2. Stem Cell Differentiation

- Advanced Cell Technology
- CardioTox
- Cellular Dynamics International
- Fate Therapeutics
- Geron
- GlaxoSmithKline
- Harvard Stem Cell Institute
- iPierian
- Novocell
- Pfizer
- Pfizer Regenerative Medicine
- Roche
- Stemgent
- VivoMedica

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