Premye done ki soti nan faz 3 HOPE-B Jijman Terapi Jij la: efikasite ak sekirite nan Etranacogene Dezaparvovec (AAV5-Padova hFIX Variant; AMT-061) nan granmoun ki gen grav oswa modere-grav Emofili B trete Irrespective of Pre-ki deja egziste anti-kapsid netralizant
Pwen esansyèl ki soti nan reyinyon 62th ASH Anyèl ak Ekspozisyon an

Premye done ki soti nan faz 3 ESPERANS-B Jijman Terapi Jij la: efikasite ak sekirite nan Etranacogene Dezaparvovec (AAV5-Padova hFIX Variant; AMT-061) nan granmoun ki gen grav oswa modere-grav Emofili B trete irrespective of pre-egziste anti-kapsid netralizant Antikò

Steven W. Pipe, MD1, Michael Recht, MD, PhD2, Nigel S. Key, MD3, Frank WG Leebeek, MD, PhD4, Giancarlo Castaman, MD5, Susan U. Lattimore, RN6, Pòl Van Der Valk7,8,9, Kathelijne Peerlinck, MD, PhD10, Michiel Coppens, MD11, Niamh O'Connell, MD, PhD12, Jan Pasi, MB, ChB, PhD, FRCP, FRCPath, FRCPCH13, Pyè Kampmann, MD14, Karina Meijer, MD, PhD15, Annette von Drygalski, MD, PharmD16, Guy Young, MD17, Cedric Hermans, MD, MRCP, PhD18, Jan Astermark, MD, PhD19,20, Robert Klamroth, MD, PhD21, Richard S. Sitwon, MD22, Natan Visweshwar, MD23, Shelley Crary, MD, MS24, Rashid Kazmi, MBBS25, Emily Symington26, Miguel A. Escobar, MD27, Esteban Gomez, MD28, Rebecca Kruse-Jarres, MD29, Adam Kotowski, MD30, Doris Quon, MD, PhD31, Michael Wang, MD32, Allison P. Wheeler, MD33, Eileen K Sawyer, PhD34, Stephanie Verweij, BSc35, Valerie Colletta, MSc36, Naghmana Bajma, MD37, Robert Gut, MD, PhD36 ak Wolfgang A. Miesbach, MD, PhD38

1University of Michigan, Ann Arbor, MI

2Oregon Sante ak Syans Inivèsite, Portland, OSWA

3Inivèsite North Carolina, Chapel Hill, NC

4Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands

5Azienda Ospedaliera Università Careggi, Florence, Itali

6Oregon Sante ak Syans Inivèsite, Portland

7Vrije Universiteit Medical Center, Amstèdam, NLD

8Inivèsite Medisch Centrum Utrecht, Utrecht, Netherlands

9Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands

10Depatman Medsin vaskilè ak Ememostazi ak Emofili Sant, Inivèsite Lopital Louvain, Louvain, Bèljik

11Sant Medikal Amstèdam Inivèsite, Inivèsite Amstèdam, Amstèdam, Netherlands

12Sant nasyonal koagulasyon, St James Hospital, Dublin, Iland

13Royal London Emofili Sant, Barts ak lekòl la London nan Medsin ak Dexter, London, Wayòm Ini

14Rigshospitalet, vil Copenhagen, Denmark

15Inivèsite Medikal Sant Groningen, Groningen, Netherlands

16Inivèsite Kalifòni San Diego, La Jolla, CA

17University of Southern California Keck School of Medicine, Children's Hospital Los Angeles, Los Angeles, CA

18Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussels, Bèljik

19Depatman Translational Medsin-klinik koagulasyon, Lund University, Malmo, Syèd

20Lopital Inivèsite Skane, Malmo, Syèd

21Vivantes Klinikum im Friedrichshain, Bèlen, Almay

22Inivèsite Utah, Salt Lake City, UT

23University of South Florida, Tampa, FL

24University of Arkansas pou Syans Medikal, Little Rock, AR

25Inivèsite Lopital Southampton NHS Fondasyon Trust, Southampton, Wayòm Ini

26Lopital Cambridge University – Addenbrooke a, Cambridge, Wayòm Ini

27Sant Syans Inivèsite Texas nan Houston, Houston, TX

28Lopital Timoun Phoenix, Phoenix, AZ

29Bloodworks Nòdwès, Seattle, WA

30Emofili Sant nan lwès New York, New York

31Los Angeles Orthopedic Hospital, Orthopedic Emophilia Treatment Center, Los Angeles, CA

32Anschutz Medical Campus, University of Colorado, Lekòl Medsin, Emofili ak Sant tronbozi, Aurora, CO

33Sant Medikal Inivèsite Vanderbilt, Nashville, TN

34uniQure Inc, Lexington, MA

35uniQure, Lexington, MA

36uniQure Inc, Lexington, MA

37Uniqure Inc, Lexington, MA

38Inivèsite lopital Frankfurt, Frankfurt, Almay

Kle Done Pwen

Imaj

HOPE-B faz 3 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 54 patisipan yo nan yon dòz 2x1013 gc / kg. Pwen esansyèl prensipal yo te aktivite FIX nan 26 ak 52 semèn apre perfusion, ak ABR nan 52 semèn konpare ak plon-an. Se dire swivi pwograme pou 5 ane. Pre-egziste antikò netralizan AAV5 pa t 'yon kritè eksklizyon.

Imaj

Figi sa a montre vle di ak medyàn FIX aktivite nan tout patisipan yo jiska 26 semèn pòs-dòz (N = 54), ak pou patisipan yo ki gen swivi pi lwen pase 26 semèn (jiska 72 semèn). Ekspresyon te gaya pa 3 semèn pòs-dòz ak mezire yon mwayèn de 37.2% nan semèn 26. Yon kantite patisipan yo gen obsèvasyon byen pi lwen pase 6-mwa final la ko-prensipal ki pa gen okenn prèv ki montre n bès nan aktivite FIX nan peryòd la swivi. .

Imaj

Tablo sa a konpare senyen pou tout patisipan yo pandan premye 6 mwa yo apre perfusion nan peryòd 6 mwa plon-an. Total senyen diminye pa 83% ak trete senyen diminye pa 91%. Pasyan ki gen 0 senyen ogmante de 30% nan peryòd plon-a 72% nan 6 mwa pòs-dòz peryòd la.

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