uniQure (QURE), CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

FREE Breaking News Alerts from StreetInsider.com!

E-mail Address

StreetInsider.com Top Tickers, 6/23/2026

1. SPCX
2. NVDA
3. ACN
4. GOOGL
5. MU
6. USO
7. APGE
8. ABBV
9. MSFT
10. IBM

Go back to uniQure (QURE), CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

December 9, 2021 7:05 AM EST

~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec ~

~ Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy ~

~ Stable and durable FIX levels with mean FIX activity of 36.9 percent of normal in full study population at 18-months, compared to a mean of 39.0 percent of normal at 6 months ~

~ Manufacturing operations supporting process validation of etranacogene dezaparvovec... More