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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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

Citirajte ovaj rad

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, i sur.. The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products. Molecular Therapy [Internet]. 12.2023. [pristupljeno 11.11.2024.]. 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, Prosinac 2023. [Online]. Dostupno na: https://urn.nsk.hr/urn:nbn:hr:184:382968. [Citirano: 11.11.2024.]

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Podaci o radu
Naslov (engleski)The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products
AutorGloria M. Palomo
AutorTomas Pose-Boirazian
AutorFrauke Naumann-Winter
AutorEnrico Costa
AutorDinah M. Duarte
AutorMaria E. Kalland
AutorEva Malikova
AutorDarius Matusevicius
AutorDinko Vitezić
AutorKristina Larsson
AutorArmando Magrelli
AutorVioleta Stoyanova-Beninska
AutorSegundo Mariz
Autorova ustanovaSveučilište u Rijeci
Medicinski fakultet
(Katedra za farmakologiju)
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Temeljne medicinske znanosti
Farmakologija
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Kliničke medicinske znanosti
Klinička farmakologija s toksikologijom
Sažetak (engleski)
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.
Ključne riječi (engleski)
Jezikengleski
Vrsta publikacijeZnanstveni rad - Izvorni znanstveni rad
Status objavePrihvaćen za objavljivanje
Vrsta recenzijeRecenziran - međunarodna recenzija
Verzija publikacijeObjavljena verzija rada (izdavačev PDF)
Naslov časopisaMolecular Therapy
p-ISSN1525-0016
DOIhttps://doi.org/10.1016/j.ymthe.2023.09.020
URN:NBNurn:nbn:hr:184:382968
Datum objave publikacije2023-12
URL dokumentahttps://linkinghub.elsevier.com/retrieve/pii/S1525001623005403
Vrsta resursaTekst
Prava pristupaOgraničen pristup
Uvjeti korištenjaIkona licence Zaštićeno autorskim pravom.
Datum i vrijeme pohrane2023-11-15 08:09:12