prikaz prve stranice dokumenta The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
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Scientific paper - Original scientific paper
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
Molecular Therapy, (2023); https://doi.org/10.1016/j.ymthe.2023.09.020
Palomo, Gloria M.; Pose-Boirazian, Tomas; Naumann-Winter, Frauke; Costa, Enrico; Duarte, Dinah M.; Kalland, Maria E.; Malikova, Eva; Matusevicius, Darius; Vitezić, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta; Mariz, Segundo

Cite this document

Palomo, G. M., Pose Boirazian, T., Naumann Winter, F., Costa, E., Duarte, D. M., Kalland, M. E. ... Mariz, S. (2023). The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products. Molecular Therapy. doi: 10.1016/j.ymthe.2023.09.020

Palomo, Gloria M., et al. "The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products." Molecular Therapy, 2023. https://doi.org/10.1016/j.ymthe.2023.09.020

Palomo, Gloria M., Tomas Pose Boirazian, Frauke Naumann Winter, Enrico Costa, Dinah M. Duarte, Maria E. Kalland, Eva Malikova, et al. "The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products." Molecular Therapy (2023). https://doi.org/10.1016/j.ymthe.2023.09.020

Palomo, G. M., et al. (2023) 'The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products', Molecular Therapy. doi: 10.1016/j.ymthe.2023.09.020

Palomo GM, Pose Boirazian T, Naumann Winter F, Costa E, Duarte DM, Kalland ME, and sur.. The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products. Molecular Therapy [Internet]. 2023 December [cited 2024 May 11]. doi: 10.1016/j.ymthe.2023.09.020

G. M. Palomo, et al., "The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products", Molecular Therapy, December 2023. [Online]. Available at: https://urn.nsk.hr/urn:nbn:hr:184:382968. [Accessed: 11 May 2024]

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Metadata
Title (english)The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
AuthorGloria M. Palomo
AuthorTomas Pose-Boirazian
AuthorFrauke Naumann-Winter
AuthorEnrico Costa
AuthorDinah M. Duarte
AuthorMaria E. Kalland
AuthorEva Malikova
AuthorDarius Matusevicius
AuthorDinko Vitezić
AuthorKristina Larsson
AuthorArmando Magrelli
AuthorVioleta Stoyanova-Beninska
AuthorSegundo Mariz
Author's institutionUniversity of Rijeka
Faculty of Medicine
(Department of Pharmacology)
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Basic Medical Sciences
Pharmacology
Scientific / art field,
discipline and subdiscipline		BIOMEDICINE AND HEALTHCARE
Clinical Medical Sciences
Clinical Pharmacology and Toxicology
Abstract (english)
In 2000, the European Union (EU) introduced the orphan pharmaceutical legislation to incentivize the development of medicinal products for rare diseases. The Committee for Orphan Medicinal Products (COMP), the European Medicines Agency committee responsible for evaluation of applications for orphan designation (OD), 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 OD, 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 EU 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.
Keywords (english)
Languageenglish
Publication typeScientific paper - Original scientific paper
Publication statusAccepted
Peer reviewPeer review - international
Publication versionPublished version
Journal titleMolecular Therapy
p-ISSN1525-0016
DOIhttps://doi.org/10.1016/j.ymthe.2023.09.020
URN:NBNurn:nbn:hr:184:382968
Publication2023-12
Document URLhttps://linkinghub.elsevier.com/retrieve/pii/S1525001623005403
Type of resourceText
Access conditionsRestricted access
Terms of useLicence In copyright icon
Created on2023-11-15 08:09:12