[Corrected, 1:25pm ET, see below] All innovation and companies start off as an idea. Some will gain more traction than others over time, but we at Xconomy think that big ideas—and the people and companies in Boston’s life sciences community bold enough to try to implement them—are worth celebrating. Some of those ideas even go against conventional thinking.
Arrakis Therapeutics – Making RNA Druggable
Drug makers have long sought to target RNA molecules, but the fast-moving, shape-changing nature of the molecules makes them tough to pin down using conventional small molecule drugs. Or so goes conventional thinking.
Arrakis Therapeutics isn’t buying this, and is intent on finding small molecules that inhibit RNA molecules linked to disease. The company is taking the established tools and techniques used by drug makers to find protein targets for small molecules, and adapting them to look for RNA targets. The goal is to find oral drugs that go after RNAs that drive cancer, neurological disease and rare genetic disorders. And the company hopes their small molecule drugs will have advantages over other ways of targeting RNA. Biologics are confined to targets on the surface of cells, and RNAi therapeutics have drug-delivery issues and have been limited to the liver and local areas of the body.
In February, Arrakis announced its $38 million Series A financing and named biotech veteran Michael Gilman as its chair and CEO.
Bridge Project – Joining Cancer Researchers Across the River
Anyone who’s lived in the Boston area knows how much of a divide the Charles River is. It slows down cross-city commutes, and can also impede scientists from different institutions across the river who might want to work together on tough research problems, such as cancer. The Bridge Project was launched in 2012 to make it easier for cancer researchers from MIT’s Koch Institute for Integrative Cancer Research in Cambridge and the Dana-Farber/Harvard Cancer Center (DFHCC) in Boston to collaborate.
Run jointly by the two institutes, the Bridge Project gives grants to teams of researchers from MIT and the Harvard-affiliated hospitals and schools. The goal is to tackle cancers that have been tough to treat, such as pancreatic and brain cancer, and to move new treatment strategies into the clinic, say Project leaders Tyler Jacks, head of the Koch Institute, and David Livingston, deputy director of the DFHCC [Editor’s note: Updated to include the names of both Bridge Project leaders].
The group has raised $35 million and awarded 37 grants to 77 labs across the Boston area. The funded research projects include developing therapeutics for pediatric brain tumors, genetic testing for cancer risk prediction, and a soon-to-begin phase 1 clinical trial of a vaccine for a specific form of lung cancer. [Editor’s note: The Bridge Project has been funding clinical research since it was founded, not just since last year as originally stated. Direct funding of clinical trials began last year].
ReadCoor – Gene Sequencing Inside Cells
Gene sequencing has steadily gotten faster and cheaper, but it generally requires that DNA and RNA be yanked out of tissues before being analyzed. ReadCoor’s technology—developed by the lab of Harvard geneticist George Church (Church is one of our judges but is not judging this category)—allows RNA to be sequenced inside individual cells and tissues so that researchers can pinpoint, in three-dimensional space, the location and identity of many RNA molecules at a time, without having to destroy cells or tissues. The technology—fluorescent in situ sequencing—could provide new insight into which genes are being turned on and where, while still preserving important features in tissue.
The company has a $2.5 million grant from the Bill & Melinda Gates Foundation to use its technology to develop new ways of detecting pathogens that cause early childhood diseases. ReadCoor is also applying its methods to cancer, to reveal new details on the genetic diversity of cells within tumors.
Sync Project – Music as Medicine
Precision medicine brings to mind drugs that target a specific form of a disease in a patient. The founders of Sync Project think precision medicine should include music. It’s a collaboration between the startup generator PureTech Health and scientists and musicians to study how music affects the body, and use that to design music-based therapeutics. (Daphne Zohar, the head of PureTech, is a judge but is not judging this category). The group aims to test these interventions in clinical trials to improve sleep and reduce stress and pain.
Andrew Lo of MIT is a finalist in this category and was covered yesterday in a story about the finalists involved in the drug pricing debate.
Chris Garabedian – A Controversial Push for FDA Approval
Depending on your point of view, Chris Garabedian was either instrumental in helping push the first approved Duchenne muscular dystrophy drug through clinical development; or complicit in a controversial and contrarian strategy to push the FDA to review a drug with flimsy data.
Garabedian, a former Gilead Sciences and Celgene dealmaker, took over Sarepta Therapeutics—then AVI Biopharma—in 2011. He nixed a lead ongoing clinical trial for the Duchenne drug eteplirsen, and instead reshaped the program around a 12-patient mid-stage trial. That study became the basis for an “accelerated” approval filing that Garabedian aggressively pursued with the FDA. Early results mobilized a vocal patient community that became a critical component to the eventual approval of eteplirsen. But agency scientists found the package of data from Sarepta to be inconclusive. Eteplirsen was only approved in September 2016 because then-FDA commissioner Robert Califf sided with top drug evaluator Janet Woodcock and overruled several dissenting colleagues. The story remains a divisive topic within biotech circles.
Garabedian’s rocky tenure at Sarepta was over by the time the FDA approval came through, but eteplirsen never would’ve been approved without him. Now he is on to another unusual venture: running an accelerator called Xontogeny that partners with entrepreneurs to help turn nascent life science technologies into biotech startups.
Tillman Gerngross – Private and Profitable Antibody Builder
Venture-backed biotech startups typically follow the same path: go public, get bought, or go bust. Tillman Gerngross’s antibody shop Adimab has gone in a completely different direction—self-sustaining, profitable, and privately held. That unusual path, combined with the in-your-face gusto Gerngross has become known for, has helped earn the outspoken Gerngross—a biochemical engineer by training, a Dartmouth professor, and a serial biotech entrepreneur—a finalist nomination on our list of contrarians.
As Adimab’s CEO, Gerngross has amassed a slew of deals for fees and recurring payments, and structured the company as an LLC so that he could distribute cash to shareholders several times over via dividends. That plan continues to pay off. The firm was reportedly worth $1 billion in 2015. And as of June 21, Adimab—now nine years old—had 50 partnerships in place with biotechs and large pharma.
Steven Pearson of the Institute for Clinical Economic Review is a finalist in the Contrarian category and was covered yesterday in a story about the finalists involved in the drug pricing debate.
Ben Fidler wrote the Contrarian profiles in this article.
This is the eighth in a series of articles profiling the Awards finalists. You can see stories on the finalists involved in the drug pricing debate, and in the Newcomer, Patient Partnership, Commitment to Diversity, Young Innovator, Startup, and CEO categories. Winners will be announced in Boston on September 26 at the Awards Gala.