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Swiv sou yon Novel Adeno-Associated Virus (AAV) Terapi Jèn (FLT180a) Reyalize Nivo Nòmal Aktivite FIX nan Pasyan Emofili B grav (HB) (Etid B-AMAZE)
Pwen esansyèl nan 14yèm Kongrè Anyèl EAHAD la

Swiv sou yon Novel Adeno-Associated Virus (AAV) Terapi Gene (FLT180a) Reyalize Nivo Nòmal Aktivite FIX nan Pasyan Emofili B grav (HB)
(Etid B-AMAZE)

Pratima Chowdary,1,2 Susan Shapiro,3 Mike Makris,4 Gillian Evans,5 Sara Boyce,6 Kate pale,7 Gerry Dolan,8 Ulrike Reiss,9 Mak Phillips,1,2 Anne Riddell,1 Maria Rita Peralta,1 Michelle Quaye,2 Ted Tuddenham,1 Andre Smith,10 Alison Long,10 Julie Krop,10 Amit Nathwani1,2

1Katharine Dormandy Emofili ak tronbozi Sant, Royal gratis Lopital, London, UK

2University College London, London, UK

3Oxford Emofili Sant, Oxford, UK

4Inivèsite Sheffield, Sheffield, UK

5Kent ak Canterbury Lopital, Canterbury, UK

6Inivèsite Lopital Southampton, Southampton, UK

7Newcastle Emophilia Comprehensive Care Center, Newcastle, UK

8Lopital St Thomas 'ak Guy a, London, UK

9St Jude Children's Research, Memphis, TN, USA

10Freeline, Stevenage, Wayòm Ini

Kle Done Pwen

Etid Design

aPrécédemment rapòte kòm 4.5e11vg / kg; bPrécédemment rapòte kòm 1.5e12vg / kg;
cPrécédemment rapòte kòm 7.5e11vg / kg; dPrécédemment rapòte kòm 9.75e11vg / kg 

Pou evalye sekirite ak efikasite nan FLT180a, yo te itilize yon rejim dòz adaptasyon, kòmanse ak pasyan 2 nan dòz ki pi ba nan 3.8 x 1011 vg / kg ak ajiste dòz ki vin apre yo optimize ekspresyon FIX pandan y ap minimize risk pou tronbozi. Dòz la pou kowòt ki sot pase a, kowòt 4, te 8.32 x 1011 vg / kg. Pwosedi repwesyon iminitè evolye pandan jijman an, sa ki lakòz yon rejim repwesyon iminitè pwofilaktik ki gen ladan tacrolimus nan kòwòt 4.

FIXE Aktivite

FIX aktivite ki baze sou tès la yon sèl-etap pou sijè yo 4 nan kòwòt 4 sou 6 premye mwa yo apre tretman ak vektè a AAVS3 ak FIX-Padova transjèn. Peak FIX aktivite te 150-250% ak ki te fèt ant 50 ak 140 jou apre-perfusion. Nan 3 nan 4 matyè yo, nivo FIX yo rete pi wo pase 100%.

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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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