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Konferans Briefings

AMT-060 Terapi jèn nan adilt ki gen emofili grav oswa modere-grav B konfime ki eksprime ekspresyon fiks ak rediksyon dirab nan senyen ak faktè IX konsomasyon pou jiska 5 an
Pwen esansyèl ki soti nan reyinyon 62th ASH Anyèl ak Ekspozisyon an

AMT-060 Terapi jèn nan adilt ki gen emofili grav oswa modere-grav B konfime ki eksprime ekspresyon fiks ak rediksyon dirab nan senyen ak faktè IX konsomasyon pou jiska 5 an

Frank WG Leebeek, MD, PhD1, Karina Meijer, MD, PhD2, Michael Coppens, MD3, Pyè Kampmann, MD4, Robert Klamroth, MD, PhD5, Roger Schutgens, MD, PhD, MSc6, Giancarlo Castaman, MD7, Erhard Seifried, MD, PhD8, Joachim Schwaeble9, Halvard Bönig1,10, Eileen KK Sawyer, PhD11, ak Wolfgang A. Miesbach, MD12

1Sant Medikal Inivèsite Erasmus, Rotterdam, Netherlands

2Depatman ematoloji, University of Groningen, University Medical Center Groningen, Groningen, Netherlands

3Sant Medikal Amstèdam Inivèsite, Inivèsite Amstèdam, Amstèdam, Netherlands

4Rigshospitalet, vil Copenhagen, Denmark

5Depatman Medsin Entèn, Medsin vaskilè ak ememostazoloji, Vivantes Klinikum im Friedrichshain, Bèlen, Almay

6Van Creveldkliniek, Inivèsite Sant Medikal Utrecht, Utrecht University, Utrecht, Netherlands

7Azienda Ospedaliera Università Careggi, Florence, Itali

8Enstiti Frankfurt, Alman Sèvis Kwa Wouj san Baden Wurtemberg-Hessen, Frankfurt, Almay

9Enstiti Frankfurt, Alman Sèvis Kwa Wouj san Baden-Wurtemberg-Hessen, Frankfurt, Almay

10Inivèsite Washington Lekòl Medsin, Seattle, WA

11uniQure Inc., Lexington, MA

12Universitatsklinikum Frankfurt, Frankfurt, Almay

Kle Done Pwen

FASE I / II DESIGN ETID

Done Pwen

Figi sa a rezime konsepsyon etid la pou faz 1/2 etid AMT-060 la. Etid la te louvri-mete etikèt sou ak eskalasyon dòz. Kowòt 1 (n = 5) te resevwa yon perfusion sèl nan yon dòz 5x1012 gc / kg ak kòwòt 2 (n = 5) te resevwa yon perfusion sèl nan yon dòz 2x1013 gc / kg. Dire swivi se 5 an pou kòwòt 1 ak 4.5 ane pou kòwòt 2.

Imaj

Pandan 5 ane (kòwòt 1) ak 4.5 ane (kòwòt 2) nan swivi, vle di andojèn aktivite FIX te 5.2% (95% CI 2.0-8.4) nan kòwòt 1 ak 7.4% (95% CI 4.2-10.6) nan kòwòt 2. FIX aktivite te dòz depandan ak ki estab pandan peryòd la swivi pou tou de kòwòt. Valè nan parantèz reprezante aktivite FIX vle di sou tan. Se sèlman valè omwen 10 jou apre dènye administrasyon FIX konsantre yo enkli.

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Sipòte pa sibvansyon edikasyon bay Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, ak uniQure, Inc.

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