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Konferans Briefings

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav: de ane done ki sòti nan yon faz 2b esè.
Pwen esansyèl ki soti nan reyinyon 62th ASH Anyèl ak Ekspozisyon an

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav: de ane done ki sòti nan yon faz 2b esè.

Annette von Drygalski, MD, Pharm D.1, Adan Giermasz, MD, PhD2, Giancarlo Castaman, MD3, Nigel S. Key, MD4,5, Susan U. Lattimore, RN6, Frank WG Leebeek, MD, PhD7, Wolfgang A. Miesbach, MD8, Michael Recht, MD, PhD9, Esteban Gomez, MD10, Robert Gut, MD, PhD11, ak Steven W. Pipe, MD12

1Depatman Medsin, Divizyon ematoloji / nkoloji, UC San Diego Sante, San Diego, CA

2Inivèsite Kalifòni Davis, Sacramento, CA

3Azienda Ospedaliera Università Careggi, Florence, Itali

4Inivèsite North Carolina nan Chapel Hill, Chapel Hill, NC

5Divizyon ematoloji, University of North Carolina nan Chapel Hill, Chapel Hill, NC

6Oregon Sante ak Syans Inivèsite, Portland, OSWA

7Sant Medikal Inivèsite Erasmus, Rotterdam, Netherlands

8Emofili Sant, Inivèsite Lopital Frankfurt, Frankfurt, Almay

9Oregon Sante ak Syans Inivèsite, Portland, OSWA

10Lopital Timoun Phoenix, Phoenix, AZ

11uniQure Inc, Lexington, MA

12University of Michigan, Ann Arbor, MI

Kle Done Pwen

Imaj

Faz 2B esè klinik la pou AMT-061 (etranacogene dezaparvovec) te fèt kòm yon louvri-etikèt, sèl etid bra. Yon sèl dòz AAV5-Padova hFIX te enfuze nan 3 patisipan yo nan yon dòz 2x1013 gc / kg. Objektif prensipal la te aktivite FIX nan 6 semèn, ak dire swivi te 5 ane. Pre-egziste antikò netralizan AAV5 pa t 'yon kritè eksklizyon.

Imaj

Pwen prensipal la nan aktivite FIX ≥ 5% nan 6 semèn pòs-perfusion te reyalize nan tout 3 patisipan yo. Aktivite FIX vle di nan 2 zan yo te 44.2%, ak aktivite FIX te estab de ane 1 a ane 2. Plis pase 2 ane, 2 oswa 3 patisipan yo pa te gen okenn senyen oswa itilizasyon konsantre FIX. Twazyèm patisipan an te gen 2 perfusion nan terapi ranplasman FIX pou 1 sispèk ak 1 konfime senyen.

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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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