Uniqure NV at Stifel CNS Days (Virtual) Transcript
Hey, good afternoon, everyone. I'm Alex Thompson, an associate here at Stifel. It's my pleasure to introduce Matt Kapusta, CEO of uniQure. To start things off, maybe, Matt, you could give a brief update on uniQure and where you're at with hemophilia and Huntington's. And then we'll jump into some more in-depth questions. So, over to you.
Yeah, sure. So I guess as of this morning, our global partner, CSL, announced the acceptance of our MAA for our hemophilia B gene therapy by EMA. So it's now formally under review in EMA, so we're very excited about that. The guidance is from CSL that they will submit the BLA in the first half of this year. So that process is moving forward.
And then for Huntington's disease, another very exciting program, we currently have a couple of Phase 1/2 studies that are currently enrolling. The first one is a randomized, controlled study -- blinded study that is taking place in the United States.
And we recently announced that we
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