Two esteemed guests—Rachel Haurwitz, PhD, and Rodolphe Barrangou, PhD—joined the State of Biotech meeting to provide a snapshot of progress in the field of CRISPR and genome editing. Haurwitz is the CEO of Caribou Biosciences, and Barrangou is a professor at North Carolina State University and the editor in chief of The CRISPR Journal.
How far has the field come in the decade since the seminal work of Doudna (with whom Haurwitz took her PhD) and Charpentier that led to the Nobel Prize? “We’re at an incredibly exciting moment in CRISPR-land,” Haurwitz said. “It’s extraordinary how quickly we’ve moved from great ideas on the whiteboard to meaningful experiments in the laboratory.” She noted that the first FDA approval for a clinical CRISPR therapy is imminent, suggesting that “we’re at a real inflection point … where CRISPR can become truly meaningful for solving challenging problems for patients.”
“We have tens of thousands of papers published by tens of thousands of authors showing that we can use that technology and deploy it to change cells precisely, accurately, and efficiently,” Barrangou added. We are entering “a critical decade” taking that technology into “the real world to tackle grand challenges with grand technologies that are ready for prime time.”
But technology development, Haurwitz said, has to be done in the context of solving a particular problem: “Not every problem is a nail that needs to be hit by a hammer. … How do you find the right tool to create the product you most desire?”
Barrangou discussed the ever-expanding CRISPR toolbox, now including transposases, Fanzors, and epigenome editing, as well as rapid progress in the development of lipid nanoparticles for gene delivery. Nevertheless, the CRISPR business sector has been suffering over the past two years.
“A lot of companies are going to be judged not by the lofty promise of their technology platforms, but by what they are able to demonstrate in the clinic in terms of potential for patients,” Haurwitz maintained. Earlier this year, Caribou released “some quite positive data” from a Phase I trial for its lead program (CB-010), which helped raise fresh capital to continue investing in the company’s programs and platform. Caribou, Haurwitz pointed out, is now “laser-focused on off-the-shelf cell therapies” for oncology indications.
Haurwitz said that she believes “we are on the cusp of a world” where patients have access to three fundamental modalities of medicines—small molecules, biologics, and “genetic medicines writ large,” with CRISPR at the vanguard.