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Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations

Splice-modulating antisense oligonucleotides (ASOs) offer treatment options for rare neurological diseases, including those with very rare mutations, where patient-specific, individualized ASOs have to be developed. Inspired by the development of milasen, the 1 Mutation 1 Medicine (1M1M) and Dutch C...

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Main Authors: Aartsma-Rus, Annemieke (Author) , Roon-Mom, Willeke van (Author) , Lauffer, Marlen (Author) , Siezen, Christine (Author) , Duijndam, Britt (Author) , Roo, Tineke Coenen-de (Author) , Schüle-Freyer, Rebecca (Author) , Synofzik, Matthis (Author) , Graessner, Holm (Author)
Format: Article (Journal)
Language:English
Published: January 20, 2023
In: RNA
Year: 2023, Volume: 29, Issue: 4, Pages: 446-454
ISSN:1469-9001
DOI:10.1261/rna.079540.122
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1261/rna.079540.122
Verlag, lizenzpflichtig, Volltext: http://rnajournal.cshlp.org/content/29/4/446
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Author Notes:Annemieke Aartsma-Rus, Willeke van Roon-Mom, Marlen Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, and Holm Graessner
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Summary:Splice-modulating antisense oligonucleotides (ASOs) offer treatment options for rare neurological diseases, including those with very rare mutations, where patient-specific, individualized ASOs have to be developed. Inspired by the development of milasen, the 1 Mutation 1 Medicine (1M1M) and Dutch Center for RNA Therapeutics (DCRT) aim to develop patient-specific ASOs and treat eligible patients within Europe and the Netherlands, respectively. Treatment will be provided under a named patient setting. Our initiatives benefited from regulatory advice from the European Medicines Agency (EMA) with regard to preclinical proof-of-concept studies, safety studies, compounding and measuring benefit and safety in treated patients. We here outline the most important considerations from these interactions and how we implemented this advice into our plan to develop and treat eligible patients within Europe.
Item Description:Gesehen am 26.04.2023
Physical Description:Online Resource
ISSN:1469-9001
DOI:10.1261/rna.079540.122

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