Updates on CRISPR therapy development and clinical trials

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When I first heard of CRISPR gene editing I was excited for my lab to try it out, but it seemed a long way from the field having even one CRISPR therapy available to treat human disease.

David Liu CRISPR Therapy
David Liu, a pioneer in CRISPR Therapy development. Image from YouTube video.

In the Knoepfler lab, we study genetic and genomic programming in human development and disease. Gene editing seemed like a great tool for us moving forward. And it has been. Of course, I’d heard of designer ZFNs and TALENs. My colleague here at UC Davis, David Segal, and his trainees had given talks on their work in that area back then.

Back in 2014 when I first started thinking more about it, CRISPR seemed a whole lot simpler.

What I didn’t imagine in those days was that there would be so many gene-editing clinical trials ongoing already in 2022. Today’s post is an update on some recent developments, both encouraging and in some ways discouraging.

There’s been some discussion lately about the possibility of CRISPR designer babies, but that’s a topic for another post. As regular readers here will know, I’ve regularly discussed that topic. You might check out my book GMO Sapiens, which is focused primarily on potential heritable use of CRISPR in people.

In this post, I’m just focused on somatic efforts.

Remarkably, I found 124 trial listings on Clinicaltrials.gov for a search for those listing either CRISPR or gene editing. Not all of them are active or interventional, but it’s still a huge number given the relative newness of this field. I’m excited about many of these trials, but the development of CRISPR sickle cell therapies is a particularly compelling area.

CRISPR trial news



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