A group of molecular surgeons aims to change the face of genomics
CEO: Katrine Bosley
Based: Cambridge, MA
Clinical focus: Gene editing
The scoop: Since the dawn of genomics, researchers have been inching toward the root of every disease, charting the roles of genes and their mutations in hopes of better treatments in the future. Incremental advances have led to lifesaving targeted therapies, exciting clinical contenders and early-stage candidates that hold great promise for sufferers of genetic diseases.
But what if, instead of treating symptoms and addressing surrogate endpoints, doctors could just go in and surgically repair misfiring genes? Thanks to some rapidly developing technology, therapeutic gene editing has gone from on-paper promise to near-clinical reality, and Editas Medicine, an early-stage startup, has united some of the field's pioneers to flesh out its potential.
What makes Editas Fierce: Editas' origin story reads like a comic book team-up. The company's 5 scientific founders are responsible for much of the published research on CRISPR, a new approach to gene editing that has grabbed headlines around the world and galvanized a new generation of work in the field. In 2013, biotech VC stalwarts Flagship Ventures, Polaris Partners and Third Rock Ventures were considering CRISPR plays all their own, but, wooed by the prestige of the Editas team, decided to unite instead, putting up $43 million to get the company off the ground. Finally, this year, biotech veteran Katrine Bosley--who made this list in 2010 with Avila Therapeutics as well as 2010's Top 10 Women in Biotech list--joined up as CEO, taking the reins as the biotech sets sail with ambitious goals in uncharted territory.
Editas' approach to gene editing centers on short, repetitive bits of genetic code and a homing protein that can shuttle them around the body. The sequences, called CRISPRs, can be imposed on any location in the genome by hitching them to the Cas9 protein, which binds to specific RNA molecules. By crafting and deploying custom CRISPRs, researchers believe they can develop a naturalistic method of correcting defective genes, getting at the underlying causes of a broad range of diseases.
The technology's potential is staggering, but, as Bosley is well aware, it's early days in the CRISPR world. Same goes for Editas, she said, which has yet to divulge much information about its plans for initial disease targets. The company is at least a few years away from clinical trials, she said, and, as has been the case for every new technological advance in biotech, gene editing is going to have its ups and downs.
"Will we face unexpected and challenging hurdles along the way? Absolutely," Bosley said. "This is drug development. We will face soul-crushing disappointments."
But Editas is prepared for the challenge, and Bosley likens the coming years of work in CRISPR to the halcyon days of monoclonal antibodies. It took decades of work to get that technology from its preclinical nascence to the tolerable, predictable treatments we know today. The same diligence is due to make CRISPR therapeutics a reality, she said, and Editas believes it has the talent and technology to do so.
"There is some future version of genome editing that is that mature and easy, and we seek to get to that point where we can apply it robustly with a lot of good knowledge as to how to do it as safely as possible and as effectively as possible," Bosley said.
In the meantime, Editas has been having early conversations with potential partners allured by the promise of gene editing, and Bosley said the biotech may well look for outside expertise as it makes its way toward the clinic. CRISPR is a methodology, not a therapeutic panacea, so while Editas has an excellent grasp of its own technology, teaming up with disease-area experts could speed the path of a promising candidate, she said.
"A lot of it is more vision than data at the moment, but everybody here knows that this is a long marathon, and everybody here is in for that marathon," she said. "... I can't tell you how long the road is, but we're here to take every step on it."
Bosley is familiar with the process of turning out-there science into practicable therapeutics. Her last company, Avila, pioneered covalent drugs, targeted therapies that form two bonds with their targets and effectively silence disease-causing proteins. But before Avila could follow through in the clinic, Celgene ($CELG) stepped in with a buyout offer worth up to $925 million, and the company's proprietary Avilomics technology is now one of the Big Biotech's many R&D tools.
Investors: Flagship Ventures, Polaris Partners and Third Rock Ventures
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Biotech star Bosley takes the helm at Editas
Biotech pioneer in 'gene editing' launches with $43M in VC cash
-- Damian Garde (email | Twitter)