From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial.

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Abstract

While iPSCs have created unprecedented opportunities for drug discovery, there remains uncertainty concerning the path to the clinic for candidate therapeutics discovered with their use. Here we share lessons that we learned, and believe are generalizable to similar efforts, while taking a discovery made using iPSCs into a clinical trial.

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Journal

Journal ID (iso-abbrev): Cell Stem Cell

Title: Cell stem cell

Publisher: Elsevier BV

ISSN (Electronic): 1875-9777

ISSN (Print): 1875-9777

Publication date (Electronic): Jul 02 2015

Volume: 17

Issue: 1

Affiliations

[1 ] Regenerative Medicine Discovery Performance Unit, GlaxoSmithKline, Cambridge, MA 02139, USA. Electronic address: john.d.mcneish@gsk.com.

[2 ] Regenerative Medicine Discovery Performance Unit, GlaxoSmithKline, Cambridge, MA 02139, USA.

[3 ] Departments of Neurology and Anesthesia, Critical Care and Pain Medicine, Massachusetts General Hospital, Boston, MA 02114, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA.

[4 ] Harvard Stem Cell Institute, Cambridge, MA 02138, USA; FM Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.

[5 ] Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Howard Hughes Medical Institute and Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Stanley Center for Psychiatric Research, Broad Institute of Harvard and MIT, Cambridge, MA 02139, USA. Electronic address: eggan@mcb.harvard.edu.

Article

Publisher Item ID: S1934-5909(15)00273-8

DOI: 10.1016/j.stem.2015.06.013

PubMed ID: 26140603

SO-VID: a5a8dd8f-3a19-4d7b-951b-939e08c1a8b3

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