Gene Editing, Venture

Eyeing Duchenne MD cure, The Column Group gambles $40M on CRISPR/Cas9 startup Exonics

The Column Group thinks that gene editing may provide just the right kind of technology needed to cure Duchenne muscular dystrophy. And the investment group is wagering $40 million that the preclinical mouse data used to back biotech startup Exonics is pointing in the right direction.

John Ripple

This infusion of cash is a big boost for Exonics, a Cambridge, MA-based company which started out with $5 million in seed money from CureDuchenne. But their backers in San Francisco are not just handing the funds off, Exonics CEO John Ripple told Endpoints News. In addition to bankrolling the preclinical research (much of which will be optimizing the current technology), it will also be pivotal in expansion efforts as Exonics looks to start a lab in Cambridge to supplement founder Eric Olson’s research at UT Southwestern.

“With Dr Olson and his team’s expertise there at UT Southwestern, and with the team that we’ve been able to build here at Exonics, and now the funding that we have, we think we’re very well positioned to at least be a leader in this field,” Ripple said, “and to hopefully be the first, or one of the first, to bring promising treatments that will address the cause of Duchenne muscular dystrophy for these boys.”

The goal is for the new lab to be operational early next year. One undisclosed scientist from Olson’s team will head the facility, and Exonics is in the process of scouting other talents, with the help of TCG’s recruitment staff.

David Goeddel

Additionally, two TCG members will be joining Exonics’ board of directors. One of them is managing partner David Goeddel, the first scientist hired by Genentech, who co-founded Tularik —a gene regulation company later acquired by Amgen. He is also serving on multiple boards including NGM Bio and Tenaya.

“The company has generated compelling early data, and we are pleased to support Exonics as it advances its preclinical development program in Duchenne closer to the clinic,” Goeddel said in a statement.

An early adopter of CRISPR/Cas-9 in treating DMD, Exonics was founded on the promise to identify and repair a range of exon mutations — not just exon 51, which FDA-approved Exondys51 targets — that prevent the production of dystrophin, the muscle fiber protecting protein missing in children with DMD.

Since Exonics launched in February, and even before, other companies have been developing their own gene therapy for DMD. Sarepta — which sells controversial Exondys51  — announced a couple weeks back that it’s partnering with Duke to explore its own CRISPR technology. This highlights the urgent unmet need for improved DMD treatment, and while Ripple acknowledges that, he said Exonics want to ensure they have it right.

“We are trying to develop an optimal treatment for these boys to make sure that we have the best possible treatment when we do enter the clinic and give that first administration to these boys,” he said.


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