QPI-1007

Nukleinsäure
Cosdosiran
Nukleoside: A = Adenosin, C = Cytidin G = Guanosin, U = Uridin, LdC = Desoxy-L-cytidin, ia = [1'-De(6-amino-9H-purin-9-yl)]desoxyadenosin = 1,2-Dideoxy-D-ribofuranose
Allgemeines
Freiname Cosdosiran[1]
Andere Namen
  • QPI-1007
Identifikatoren
CAS-Nummer 1834560-88-1
Wirkstoffdaten
Wirkmechanismus RNA-Interferenz
Eigenschaften
Größe
Struktur Synthetisches doppelsträngiges siRNA-Oligonukleotid

QPI-1007 (internationaler Freiname Cosdosiran) ist ein experimenteller Arzneistoff aus der Gruppe der RNAi-Therapeutika zur Behandlung der nichtarteritischen anterioren ischämischen Optikusneuropathie.

Eigenschaften

QPI-1002 ist eine RNA, die an die mRNA von Caspase 2 bindet und durch RNA-Interferenz zu einem Gen-Knockdown der Caspase 2 führt.[2] Es befand sich im Jahr 2017 in klinischen Studien der Phase 3.[3] Die Applikation erfolgt durch Injektion in den Glaskörper.[2]

Einzelnachweise

  1. ↑ INN Recommended List 78, World Health Organisation (WHO), 9. September 2017.
  2. ↑ a b Jens Kurreck: Molecular Medicine. John Wiley & Sons, 2016, ISBN 978-3-527-33189-5, S. 323.
  3. ↑ R. Titze-de-Almeida, C. David, S. S. Titze-de-Almeida: The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market. In: Pharmaceutical research. Band 34, Nummer 7, Juli 2017, S. 1339–1363, doi:10.1007/s11095-017-2134-2, PMID 28389707.

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