fig2

2017-2023: state of the art of gene therapies in rare diseases in Europe: the dynamics of clinical R&D, new approved treatments and expected therapies in the pipelines

Figure 2. General distribution and temporal dynamic of clinical studies related to gene therapies in rare diseases in Europe from GENOTRIAL database. (A) Distribution of clinical trials considered as «Gene therapies» in EudraCT database from 01/01/2017 to 01/01/2023 (n = 650) vs. other types of clinical trials not considered as gene therapies (n = 12.648); (B) Distribution of clinical trials considered as « Gene therapies in rare disease» in EudraCT database from 01/01/2017 to 01/01/2023 (n = 300) vs. other trials Gene therapy (n = 350); (C-D) Distribution of Clinical trial phases from phase I to phase III, respectively, among (C) the identified 300 country-specific clinical trials or (D) their 93 associated clinical studies related to gene therapies in rare diseases from the constructed database; (E) Representative time charts of the number of clinical studies, including gene therapies, started between 2011 and 2022 (n = 93); (F) Distribution of current status of identified Phase I-III clinical studies, "ongoing", "completed", or "prematurely ended" (n = 93); (G-H) Representative time charts of the number of clinical studies including gene therapies (G) having been completed between 2017 and 2022 and started between 2011 and 2022, (n = 22) or (H) being currently still ongoing as of 01/01/2023 and having started between 2013 and 2022 (n = 67).

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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