First U.S. patient treated with base-editing therapy
More precise and possibly powerful than CRISPR
Havard professor David Liu, the discoverer of base editing and one of the founders of Beam Therapeutics
What's the news: The first patient in the U.S. has received base-editing therapy.
Why should we believe it: This news is based on a press release earlier this week by Beam Therapeutics. Beam is a biotech company founded by several pioneers in gene editing, among them Harvard professors David Liu and Keith Joung.
According to the press release, a patient was treated in August with BEAM-201, a gene editing-based therapy for relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma.
Why this is a big deal: This is only a phase 1 study to test the safety and viability of this new treatment. But it's the precedent that's important, and the future it points to.
Base editing is an exciting “next-generation” form of gene editing. While other forms of gene editing have been compared to a pair of scissors because they require double strand breaks, base editing works more like a pencil with an eraser, and can make specific changes to DNA.
Base editing was only discovered in 2016. The first base-editing treatment in a human happened earlier this year in the UK. The announced Beam research is the first in the US. It shows how quickly this field is developing, and points the way forward.
So what specifically can you do now: If by chance you know someone suffering from acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma, Beam's clinical trial is now open for enrollment. And if you're wondering where this all might be headed, read on to find out more about the transformative, alarming power of gene therapy.