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- W4387340881 abstract "In the year 2000, the European Union (EU) introduced orphan pharmaceutical legislation to incentivise the development of medicinal products for rare diseases. The Committee for Orphan Medicinal Products (COMP), the European Medicines Agency (EMA) committee responsible for evaluation of applications for orphan designation, received an increasing flow of applications in the field of gene therapies over the last years. Here, the COMP has conducted a descriptive analysis of applications regarding gene therapies in non-oncological rare diseases, with respect to a) targeted conditions and their rarity b) characteristics of the gene therapy products proposed for orphan designation, with a focus on the type of vector used and c) regulatory aspects pertaining to the type of sponsor and development, by examining the use of available frameworks offered in the European Union such as protocol assistance and PRIME. It was noted that gene therapies are being developed by sponsors from different backgrounds. Most conditions being targeted are monogenic, the most common being lysosomal disorders, and with a very low prevalence. Generally, adeno-associated viral vectors were being used to deliver the transgene. Finally, sponsors are not frequently using the incentives that may support the development and the reasons for this are unclear." @default.
- W4387340881 created "2023-10-05" @default.
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- W4387340881 date "2023-10-01" @default.
- W4387340881 modified "2023-10-06" @default.
- W4387340881 title "The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products" @default.
- W4387340881 doi "https://doi.org/10.1016/j.ymthe.2023.09.020" @default.
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