Broad Institute president Eric Lander kicked things off talking about the interconnectedness of the Boston biotech ecosystem. It’s critical that all of it---academia, venture firms, and scientific entrepreneurs---continues to thrive. Otherwise, “the whole thing shrivels up,” he said.
This fellow (middle) wins the day’s award for most perfectly arranged pocket square.
Insurers have been preparing for the arrival of gene therapy, but a lot of work has to be done for the U.S. healthcare system to successfully adapt. Harvard Pilgrim Health Care chief medical officer Michael Sherman (2nd from right) says we “need to solve for the budget impact” of gene therapies as more and more come to market, or risk situations in which patients who should get treated don’t get access.
Michael Sherman and Alnylam Pharmaceuticals president Barry Greene (right) getting a jump on discussing pricing models for Alnylam’s soon-to-be-approved RNA interference medicine, patisiran.
Remember to bring questions! We always leave some time for a few from the audience.
The Institute for Clinical and Economic Review (ICER) worked with Spark for two years to discuss Luxturna’s value before it was approved, but their views ultimately still differed. That speaks to ICER’s growing role as an independent drug pricing arbiter. “While it’s great that companies are recognizing there will be potentially difficult, intense value conversations, that doesn’t let them off the hook in terms of evidence generation,” said ICER's Dan Ollendorf (far left).
Goldfinch Biopharma scientific founder Anna Greka (right) and Foghorn Therapeutics founder Cigall Kadoch having a laugh before their panel.
From left: Anna Greka, Amy Schulman, Cigall Kadoch, and moderator Teresa Lavoie discussed the ups and downs of trying to be an academic-turned young scientific founder. These days, the pressure is on academics to translate ideas more than before. “We joke that sometimes with faculty, unless you make a startup you can’t get promoted,” Greka said.
Progress is being made in increasing the ranks of female scientific founders and executives, but there is a long way to go, said Amy Schulman. One key to improving gender equality in the life sciences: directly acknowledging and challenging unconscious bias when it rears its head.
Dana-Farber clinicians have learned plenty of lessons about which patients might be best suited for CAR-T treatment and how they’ll respond, said oncologist Caron Jacobson. But the neurological side effects it can cause are less understood, and can be alarming to deal with. Some patients have trouble speaking, or seem like “nobody’s home.” “It’s tricky to get patients through this,” she said.
Tufts Medical Center, meanwhile, has yet to treat a commercial patient with CAR-T, said Andreas Klein. Tufts has been assembling the team and preparing doctors for the myriad issues that can arise, but it has taken time to figure out how it will absorb the costs of the pricey therapies. “Who is going to write that check?” he said.
Gene editing pioneer Feng Zhang noted that important work is being done to try to optimize CRISPR-based treatments so they can properly address diseases without causing unintended effects. “The FDA is working closely with drug developers to figure out the best way to bring the technology forward, but in a thoughtful way,” he said. “It’s a community effort.”
Craig Mello (left) co-discovered RNA interference some two decades ago. With RNAi on the verge of its first approved drug and CRISPR just starting its first human clinical tests, it’s worth remembering the ups and downs RNAi had to go through. “CRISPR is where RNAi was 15 years ago,” Mello said.
It took “about a decade and for us, about $1 billion” to figure out how to deliver large RNAi molecules safely and effectively into the liver, Alnylam's Barry Greene said. Both he and Craig Mello said the next frontier for RNAi therapeutics is delivering them to the brain. “We and academia are starting to see some positive signs” that it’s possible do that, Greene said.
Nobel laureate Craig Mello (right) amongst those sticking around for drinks after the show.
Cigall Kadoch possibly talking about the size of Foghorn’s first financing round.
CAR-T. Gene therapy. RNA interference. These aren’t just science experiments. They’re real, cutting-edge medicines either just on the market or soon to be. And the implications of their arrival was the focus of a few spirited discussions at Xconomy’s “What’s Hot in Boston Biotech” event last week at the Broad Institute in Cambridge, MA.
Today, we’re checking back with a small taste of the festivities through a slideshow and some takeaways, but first some thank-yous to everyone who made the event possible:
Thanks to our attendees and speakers. Thanks also to our event host, the Broad Institute, and our sponsors Fish & Richardson, ICON, Sanofi, and Ten Bridge Communications. And a hat tip to Keith Patankar for the pictures.
With that, hope you enjoy the photos and see you again next time!