Editas bags CRISPR tech from MGH to add therapeutic breadth to pipeline

DNA

Editas Medicine ($EDIT) has licensed Cas9 nuclease and PAM variant technology from Massachusetts General Hospital (MGH). The exclusive licensing deal sees Editas return to the lab of its cofounder for IP it thinks can improve specificity and expand the therapeutic breadth of its R&D operation.

A team overseen by Keith Joung, a scientific cofounder of Editas, touted the potential of the Cas9 technology in papers published in Nature and Nature Biotechnology over the past year. The most recent paper, which was released in January, described the advance Editas sees improving specificity, the high-fidelity Cas9 variant Streptococcus pyogenes Cas9-HF1. This cut levels of off-target effects to the extent that they became undetectable, prompting Joung to claim it would become the go-to Cas9 for many applications.

The other, earlier papers discussed technologies Editas thinks will enable it to go after a wider range of genetically defined diseases. In this work, the MGH team identified S. pyogenes and S. aureus Cas9 PAM variants. PAM, an acronym of protospacer adjacent motif, is the section of Cas9 that dictates where the protein binds to DNA. With these tools potentially broadening the number of sites Editas can target for editing, it could enable it to pursue diseases that were beyond the reach of its first-generation capabilities.

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“This agreement with MGH marks additional progress on our strategy of building a company committed to advancing the science behind CRISPR to benefit patients facing genetically-defined diseases,” Editas CEO Katrine Bosley said in a statement. “These advancements align fully with our highly differentiated genome editing platform. We are eager to deploy them and unlock their therapeutic potential.”

The licensing deal with MGH, which Editas tapped for tech back in 2014 to get its R&D operation started, is indicative of the ongoing arms race in genome editing technology. Editas has established itself at the forefront of this fight, in part because of its links to MGH. But the biotech isn’t relying on these ties to maintain its position. Last week, Editas reached across the Atlantic for access to know-how, striking a three-year deal with renowned Italian research center the San Raffaele Telethon Institute for Gene Therapy.

- read the release

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