Base Editing for Therapeutics: John Evans on The Long Run

John Evans is today’s guest on The Long Run.

John is the CEO of Cambridge, Mass.-based Beam Therapeutics. The company was founded a little over three years ago to develop several types of DNA base editing developed in David Liu’s lab at Harvard University, along with an RNA base editor platform developed by Feng Zhang, at the Broad Institute of MIT and Harvard.

The gist of the idea, which John describes well, is that it’s possible to make precise gene edits with an adapted form of CRISPR technology that doesn’t need to cut out a whole disease-related gene. It can achieve a therapeutic goal by acting in an even more precise way – by finding an exact spot in the 3-billion base pair genome, and modifying a single chemical base of DNA.

Beam is seeking to put different pieces of the technology puzzle together, with different types of editing, different modes of delivery, and thinking through how these combinations make the most sense for different therapeutic indications. When does it make sense to do edits outside the body (ex vivo), and when it’s better to do the edit inside the body (in vivo)?

The company’s first two programs aimed for the clinic, BEAM 101 and BEAM 102, are for sickle cell disease and beta-thalassemia.

It’s heady stuff. As it matures and becomes closer to becoming a real thing that might become an FDA approved product, it’s likely to generate a lot of fear and controversy in the public. I asked John a bit about that toward the end of the conversation.

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Now, please join me and John Evans on The Long Run.