Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.
Sign Up to like & getrecommendations! Published in 2019 at "Blood advances"
DOI: 10.1182/bloodadvances.2019000811
Abstract: Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter. A phase 2b study was conducted to confirm that… read more here.
Keywords: activity; cessation; dezaparvovec amt; fix activity ... See more keywords
Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy.
Sign Up to like & getrecommendations! Published in 2022 at "Blood advances"
DOI: 10.1182/bloodadvances.2022008886
Abstract: Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene with a liver-specific promoter. Here we report 3-year outcomes from a Phase 2b,… read more here.
Keywords: year; etranacogene dezaparvovec; factor; fix ... See more keywords