This Scientist Is Building Custom Gene-Editing Tools—And Stands To Make Billions

Benjamin Oakes’ Scribe Therapeutics is developing specialized Crispr proteins to tackle a wide range of diseases–and it’s garnered deals with Big Pharma potentially worth over $4 billion.

In 2013, Benjamin Oakes was hellbent on getting his PhD while working on the bleeding edge of molecular engineering: refining a gene editing tool, Crispr, that promised to some day cut DNA as precisely as a pair of scissors. There were two leading research groups at the time — one at the University of California, Berkeley, led by future Nobel Prize winner Jennifer Doudna, the other at the Broad Institute jointly run by Harvard and MIT — and Oakes was vacillating endlessly between them. Which is how he found himself one day in Doudna’s house, part of a gathering of promising students being considered to work in Berkeley’s labs.

There, he met David Savage, then a professor at Berkeley who had just started his own lab focused on engineering proteins like ones used in Crispr systems. Oakes had interviewed to join Savage’s lab, too, but in the more relaxed setting they geeked out over the potential of new tools in the space accurate enough to essentially cut a function from one protein and paste it into another. Not long after, Oakes solved his career conundrum: he joined both labs, where his research focused on improving the gene-editing potential of Crispr by making protein engineering tools more customizable and controllable.

Ten years later, he’s applying that work in a company he cofounded with Doudna and Savage, Scribe Therapeutics. The company is building a biological platform of customized gene-editing tools to tackle a wide range of hard-to-treat diseases from ALS to cancer to sickle cell anemia. It’s backed by over $120 million in venture investments from major firms like Andreessen Horowitz and OrbiMed — and it already has partnerships with major pharmaceutical companies potentially worth over $4 billion dollars.

The story of Crispr starts with bacteria, whose immune systems evolved to attack invading viruses by cutting up crucial parts of their DNA. This discovery was first applied to gene editing in combination with a special class of bacterial proteins called Cas9. The potential for this technology is enormous: it makes it possible to consider curing genetic disorders with a one-and-done treatment. But it’s not without complications. Because viruses mutate, Crispr systems aren’t completely precise, creating a risk that the wrong part of someone’s DNA might be cut by a gene-editing system.

A little over a decade since its discovery, the promise of Crispr has already begun to be realized with viable applications in agriculture as well as diagnostic testing. Last April, a collaboration of Vertex Pharmaceuticals and Crispr Therapeutics filed the first full FDA application for approval of a Crispr/Cas-9 gene editing treatment for patients with sickle cell anemia. The treatment showed strong results in clinical trials with a stunning 94% of patients treated hitting the desired outcomes. The FDA is expected to make a decision on approval before the end of the year. Other Crispr-derived therapeutics for type one diabetes and multiple types of cancer are in the pipeline.

Oakes, 34, is already working on the next generation of the technology. His company uses a different set of proteins for Crispr systems, called “CasX”, that were discovered by Doudna’s research group. Scribe has developed a platform with CasX it calls “Crispr-by-design” that enables the company to tackle multiple types of disease. Its primary focus is enabling gene editing therapies to be delivered directly to a patient (“in vivo”) instead of removing cells from the body, editing the genes, and returning them. For example, the Vertex treatment for sickle cell involves removing stem cells from patients’ bone marrow, editing them, and returning them to the patient after they’ve undergone chemotherapy to eliminate the non-edited stem cells…







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