Commercialization challenges associated with induced pluripotent stem cell-based products
Abstract
Induced pluripotent stem (iPS) cells have generated excitement in the regenerative medicine industry. Products derived from iPS cells could be used in a range of drug discovery and development processes. These nontherapeutic products will continue to be launched over the next 5 years, and provide income and knowledge to drive the therapeutic use of iPS cells forward. While the commercial opportunity for iPS cell-based therapies is potentially large, the looming technical and scientific hurdles must be overcome and, thus, the launch of a therapy based on iPS cells is unlikely to occur until the 2020s. While the launch of a therapeutic is many years away, the business models for commercialization should be well understood and proven based on experience with other non-iPS cell-based therapies (both autologous and allogeneic) that will already be on the market.
Papers of special note have been highlighted as: ▪ of interest ▪▪ of considerable interest
Bibliography
- 1 Mason C: Regenerative Medicine 2.0. Regen. Med.2(1),11–18 (2007).
- 2 Plagnol AD, Rowley E, Martin P, Livesey F: Industry perceptions of barriers to commercialization of regenerative medicine products in the UK. Regen. Med.4,549–559 (2009).▪ An interesting analysis of barriers to commercialization based on interviews performed in the UK. The findings are directly applicable to most other regions of the world.
- 3 Mason C, Manzotti E: Induced pluripotent stem cells: an emerging technology platform and the Gartner hype cycle. Regen. Med.4,329–331 (2009).
- 4 Takahashi K, Yamanaka S: Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell126,663–676 (2006).▪▪ First paper that documented the creation of induced pluripotent stem (iPS) cells and is a must read in the iPS arena.
- 5 Takahasi K, Tanabe K, Ohnuki M et al.: Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell131,861–872 (2007).
- 6 Oikita K, Ichisaka T, Yamanaka S: Generation of germline competent induced pluripotent stem cells. Nature448,313–317 (2007).
- 7 Gunaseeli I, Doss MX, Antzelevitch C, Hescheler J, Sachinidis A: Induced pluripotent stem cells as a model for accelerated patient- and disease-specific drug discovery. Curr. Med. Chem.17(8),759–766 (2010).
- 8 Shi Y: Induced pluripotent stem cells, new tools for drug discovery and new hope for stem cell therapies. Curr. Mol. Pharmacol.2,15–18 (2009).
- 9 Kiskinis E, Eggan K: Progress toward the clinical application of patient-specific pluripotent stem cells. J. Clin. Invest.120,51–59 (2010).▪▪ Excellent review of the applications of iPS cells as both therapies and as R&D enabling tools.
- 10 Rolletschek A, Wobus AM: Induced human pluripotent stem cells: promises and open questions. Biol. Chem.390,845–849 (2009).
- 11 Feng Q, Lu S-J, Klimanskaya I et al.: Hemangioblastic derivatives from human induced pluripotent stem cells exhibit limited expansion and early senescence. Stem Cell28(4),704–712 (2010).
- 12 Das AK, Pal R: Induced pluripotent stem cells (iPSCs): the emergence of a new champion in stem cell technology-driven biomedical application. J. Tissue Eng. Regen. Med. doi:10.1002/term.258 (2010) (Epub ahead of print).
- 13 Baker M: Testing time for stem cells. Nature463,719 (2010).
- 14 Ledford H: In search of a viable business model. Nat. Rep. Stem Cells2(3),351–374 (2007).
- 15 Rowntree RK, McNeish JD: Induced pluripotent stem cells: opportunities as research and development tools in 21st century drug discovery. Reg. Med.5(4),557–568 (2010).
- 16 Kettenhofen R, Bohlen H: Preclinical assessment of cardiac toxicity. Drug Discov. Today13,702–707 (2008).
- 17 Gintant G: Pre-clinical cardiac toxicity: testing compounds beyond hERG. Int. Drug Discov.3(5),12–19 (2008).
- 18 Lee WM: Drug-induced hepatotoxicity. N. Engl. J. Med.349,474–485 (2003).
- 19 Cornwell PD, Ulricht PD: Investigating the mechanistic basis for hepatic toxicity induced by an experimental chemokine receptor 5 (CCR5) antagonist using a compendium of gene expression profiles. Toxicol. Pathol.35,576–588 (2007).
- 20 Webb S: The gold rush for induced pluripotent stem cells. Nat. Biotechnol.27,977–979 (2009).▪▪ Good review of the state of the iPS field including challenges facing intellectual property.
- 21 McBride R: Stem Cell firms shift from treatments to tools. The Boston Globe, 5 June (2008).
- 22 Barthelemy N: Investor presentation. Presented at: Life Technologies Investor Day. Cell Systems, Carlsbad, CA, USA, 2 June 2010.
- 23 Smith D: Creating partnerships with large pharma? getting ready for the big day. In: The World Stem Cell Report 2009. Siegal B (Ed.). Genetics Policy Institute, FL, USA 131–137 (2009).
- 24 Smith D: Successful business models for cell-based therapies. In: The World Stem Cell Report 2008. Siegal B (Ed.). Genetics Policy Institute, FL, USA 158–162 (2008).
- 25 Martin P, Brown N, Turner A: Capitalizing hope: the commercial development of umbilical cord blood stem cell banking. New Genet. Soc.27,127–143 (2008).
- 101 Cellular Dynamics International www.cellulardynamics.com
- 102 Cellartis www.cellartis.com
- 103 Vistagen www.vistagen.com
- 104 iPierian www.izumibio.com
- 105 Fate Therapeutics www.fatetherapeutics.com
- 106 Stemgent www.stemgent.com
- 107 MHT: Stemgent, Fate Therapeutics launch stem cell platform www.masshightech.com/stories/2009/04/20/daily39-Stemgent-Fate-Therapeutics-launch-stem-cell-platform-.html
- 108 ArunA Biomedical, Inc. http://arunabiomedical.com/index.php
- 109 Drug Discovery News. Cellular Dynamics International reprograms blood cells into stem cells using a small volume of human blood www.drugdiscoverynews.com/index.php?newsarticle=3129
- 110 Free Press Release. The market size of stem cell research products is expanding through double digit growth www.free-press-release.com/news/200902/1235794980.html
- 111 Ubiquigent: Stemgent invests $4.5 million, announces formation of ubiquigent, a new UK-listed biotechnology company based in Dundee, Scotland www.ubiquigent.com/stemgent_ubiquigent_release.php
- 112 Life Technologies www.lifetechnologies.com/about-life-technologies/history.html▪ Invitrogen acquired 15 companies in 3 years as it bought up small companies to bolster its product portfolio
- 113 Geron www.geron.com
- 114 ADIS Insight www.adisinsight.com
- 115 Cytori www.cytoritx.com
- 116 Genzyme (see annual report) www.genzyme.com
- 117 Viacord www.viacord.com
- 118 CHDI Foundation www.highqfoundation.org
- 119 Juvenile Diabetes Research Foundation International www.jdrf.org
- 120 Advanced Cell Technology www.advancedcell.com
- 121 BD www.bd.com
- 122 Biotime, Inc. www.biotimeinc.com
- 123 GlaxoSmithKline www.gsk.com
- 124 International Stem Cell Corporation www.internationalstemcell.com
- 125 Pfizer www.pfizer.com
- 126 Medtronic www.medtronic.com
- 127 Boston Scientific www.bostonscientific.com
- 128 Consumer Watchdog www.consumerwatchdog.org/patients/articles//?storyId=13759&bIndex=5
- 129 Product Launch PR www.productlaunchpr.com/PRODUCTLAUNCHPR.COM/Pharmaceutical_Consulting_%26_Sales_in_Puerto_Rico.html
- 130 PAREXEL www.parexel.com