Molecular Therapy - Methods & Clinical Development

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Molecular Therapy - Methods & Clinical Development Clinical development of gene- and cell-based therapies: overview of the European landscape  Sofieke de Wilde, Henk-Jan Guchelaar, Maarten Laurens Zandvliet, Pauline Meij  Molecular Therapy - Methods & Clinical Development  Volume 3, (January 2016) DOI: 10.1038/mtm.2016.73 Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy Terms and Conditions

Figure 1 Gene- and cell-based therapy landscape. CTMP, cell therapy medicinal product; GTMP, gene therapy medicinal product; SME, small and medium-sized enterprise; TEP, tissue-engineered product. (a) Distribution of the indication areas for the gene- and cell-based therapy, showing absolute numbers derived from the public domain of the EudraCT database*; (b) Absolute numbers of clinical trials with the of gene- and cell-based therapy product types per year; (c) Numbers (represented in percentages, n = 278) of different clinical trials study phases performed with gene- and cell-based therapy products. * Since phase 1 clinical trials are not in the public domain of EudraCT and processing of trials in EudraCT can be delayed, clinical trials may be missing. Molecular Therapy - Methods & Clinical Development 2016 3, DOI: (10.1038/mtm.2016.73) Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy Terms and Conditions

Figure 2 Country-specific variations in gene- and cell-based therapy development. (a) Percentage of clinical trials with gene- and cell-based therapies per country derived from the public domain of the EudraCT database* depicted in color and absolute numbers of clinical trials performed per country (written in country). (b) Ratio of the number of clinical trials with gene- and cell-based therapy products performed in a country per the total amount of clinical trials in that country derived from the public domain of the EudraCT database. The white colored countries did not perform any studies with gene- and cell-based therapy products or do not belong to the European Union. (European map adjusted from http://www.youreuropemap.com/). * Since phase 1 clinical trials are not in the public domain of EudraCT and processing of trials in EudraCT can be delayed, clinical trials may be missing. Molecular Therapy - Methods & Clinical Development 2016 3, DOI: (10.1038/mtm.2016.73) Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy Terms and Conditions

Figure 3 Sponsor type variations in gene- and cell-based therapy development. CTMP, cell therapy medicinal product; GTMP, gene therapy medicinal product; SME, small- and medium-sized enterprise; TEP, tissue-engineered product. (a) Distribution of the different sponsors that performed clinical trials with gene- and cell-based therapies. (b) Absolute numbers of clinical trials performed with gene- and cell-based therapy types per sponsor and the total absolute number of clinical trials with the different gene- and cell-based therapy types. (c) Absolute numbers of clinical trials in different clinical phases per sponsor and the total number of clinical trials per clinical phase. (d) Starting material per sponsor type. Molecular Therapy - Methods & Clinical Development 2016 3, DOI: (10.1038/mtm.2016.73) Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy Terms and Conditions

Figure 4 Database record selection and arrangement. (a) A schematic overview of the EudraCT data record selection. Trial data records from the EudraCT queries were reduced towards product records with corresponding clinical trials. (b) A schematic overview on how the database ProMISe was built, by coupling of clinical trials to the product and coupling of the countries in which the clinical trials have been performed. Molecular Therapy - Methods & Clinical Development 2016 3, DOI: (10.1038/mtm.2016.73) Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy Terms and Conditions