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scaled commercially—so folks scattered across the country can use it, not just a few people at a major academic center?
Wilson cautioned it’s taken three decades of work on engineering cell therapies—the first CAR was made in 1989—and the viral vectors used to deliver them, just to get to the point that even a few people have seen dramatic results. Patience, and more tinkering, is required. “The advantage right now is that in many ways we have all of those pieces in place,” he said. But “as we go into solid tumors, additional kinds of engineering [and] modifications are going to be useful in seeing the potential for these kinds of therapies…and new technologies like gene editing may have the potential to enable some of those modifications.”
4. Boston biotech’s biggest challenge? Scale. Noubar Afeyan remembers making the trek out to San Francisco “with envy,” seeing how the Bay Area first dominated the computer industry and then became a biotech powerhouse. But Boston (“and by Boston I really mean Cambridge,” he joked), has surged to become arguably a bigger biotech power, something Afeyan said was due to a sea change in attitude—more courage to pursue ideas even if they might not work.
The biggest worry in maintaining this momentum? Scale. Rent prices have surged to $90-$100 per square foot, there’s no affordable lab space “within a functioning T distance” (Boston’s train system). That’s an issue the area is going to have to confront if the good times are to continue. “I don’t think Boston will remain the Silicon Valley of biotech if it doesn’t continue to scale,” Afeyan said. “We need to think ahead about what maintains this, what makes this sustainable.”
5. Gene therapy’s limitations, and its “left field” enemy. Despite gene therapy’s re-emergence, and all the technological advances that have been made (particularly in how these therapies are delivered), Olivier Danos and Steve Paul warned of its limitations; Paul called gene therapy a “resurgent old field” that is still “in its early days.” It’s very limited; gene therapy can’t tackle difficult diseases—diabetes, for instance—and a number of advances will have to be made before it can. Those delivery systems, or vectors, are much better than they used to be, but are still evolving—“we’ll be talking about very different vectors 5 or 6 years from now,” Paul said.
But what if, by the time those vectors are ready, gene therapy doesn’t have the same audience? Kolchinsky theorized, for instance, that if carrier testing—genetic tests for prospective parents—becomes commonplace, perhaps the number of people born with monogenic disorders (the supposedly easier targets for gene therapy) will shrink. And perhaps that will force gene therapy companies to evolve, and try to crack harder, polygenic targets.
6. Deals are really about relationships. In a way, Vedanta Biosciences and Johnson & Johnson gravitated towards one another. The startup is developing microbiome therapies for immunology, and that’s right in J&J’s wheelhouse. But the deal the two cut in January was a result of personal relationships, not just strategic alignment. For one, Vedanta’s founders at PureTech began talking to J&J before the company was even incorporated. That familiarity came in handy when J&J opened up its Boston Innovation Center in 2013. While the center was being built, its Boston team “needed a place to crash,” said PureTech partner and Vedanta chief operating officer Bernat Olle, “so they took some offices at PureTech—and the relationship became hyperlocal.”
That gave J&J an important inside track. As Vedanta moved forward, it was approached by other VC groups aiming to form a syndicate, and then pharmaceutical companies looking to make a deal. Vedanta chose a licensing deal with J&J—in part because it knew the people involved, and what they wanted to do with its lead drug. “When you’re sharing your baby, all these considerations come to mind, but knowing what was in the mind of the people that were going to drive the project forward was valuable,” Olle said.
7. Give “Precision Medicine” (a lot of) time; it just might change drug R&D. President Obama’s Precision Medicine Initiative sounds great in theory; through evolving science, genomic understanding, and data collection, someday we’ll all get the drugs that specifically target the genetic problems that cause our diseases, and that starts with matching specific genetic defects with disease with the help of a massive database. But as David Altshuler said, that’s only step one, which, “while foundational and vastly important,” leads to understanding, not medicines. Step two is discovering a therapeutic, which takes another three to five years; step three, developing and testing it, takes another 10 years and hundreds of millions of dollars.
All of which is why Altshuler bristles at the idea that genomics hasn’t delivered the goods because R&D productivity rates for drugmakers remains low.
“We’re just now entering the era where we understand disease well enough,” he said. “We’ll learn over the next 10 or 20 years what the true success rates can be when you have a human biologically-focused, genetically enabled therapeutic strategy.”