ICER, CRISPR, Biotech Activism & More: The X of the Year Finalists

Xconomy Boston — 

With so many stellar Xconomy Award nominees this year, we wanted to recognize several who don’t fit neatly into the other categories, so we’ve created categories just for them. One of these nominees for X of the Year is gaining influence in the drug industry with its evaluations of drug cost effectiveness. Two more been pushing their fellow biotech CEOs to be more politically outspoken. Yet another finalist is bridging the global scientific divide by bringing lab equipment to researchers in the developing world. Read on for more details on the X of the Year finalists.

(This is the final article profiling the finalists for the Xconomy Awards. Read about the finalists in our other categories here: CEOStartupDigital TrailblazerInnovation at the IntersectionBig IdeaContrarianNewcomerYoung InnovatorCommitment to Diversity and Patient Partnership. We have also announced the winner of our Lifetime Achievement Award. All other winners will be announced at the awards gala on Sept. 5 in Boston.)

Nina Dudnik – Global Science Supporter
For more than a decade, Nina Dudnik has been building bridges in science between the developed and developing worlds. The nonprofit she founded and leads, Seeding Labs, has brought new and gently used lab equipment and other resources from 140 partners in academia and industry to more than 50 research institutions and universities in 29 countries in Africa, Latin America, and Asia. At Garden City University College in Ghana, more than 1,500 students are using three tons of lab equipment—including incubators, refrigerators and microscopes—that Seeding Labs collected, tested, and then shipped over from organizations such as Merck, Takeda and Thermo Fisher. The students are using the equipment to train to become nurses, scientists, and lab technicians.

Dudnik started building Seeding Labs while still a molecular biology graduate student at Harvard Medical School in 2003. Before her time at Harvard, she did research on plant and rice genetics in Italy and Cote d’Ivoire.

Anna Greka – Translational Researcher
Anna Greka’s role as a physician-scientist at Brigham and Women’s Hospital was critical in the discovery of a potential new treatment for patients with a rare kidney disorder. When a colleague told her about CD80, a molecular target in diseased kidney cells, it rang a bell with Greka—a drug approved for rheumatoid arthritis also acts on this target. Greka treated a patient of hers with the drug, abatacept, and saw remarkable results. She and her colleagues published their findings: five patients had partial or complete remission of a kidney disease called focal segmental glomerulosclerosis. Greka is now involved in a 90-patient clinical trial testing abatacept in people with kidney disease.

More recently, Greka and her lab discovered a small molecule that prevented kidney cell loss in animal models of kidney disease.  She also co-founded Goldfinch Bio, which is developing targeted drugs for kidney disease.

Steve Holtzman & Jeremy Levin – Activists
After the violent and deadly rally and protests in Charlottesville, VA, last August, Decibel Therapeutics CEO Steve Holtzman, and Ovid Therapeutics CEO Jeremy Levin penned an op-ed that called on fellow biotech leaders to be more politically outspoken. They asked whether staying silent on issues ranging from racism to immigration and healthcare reform—to win a seat at the table to discuss federal policy and regulation—was jeopardizing the “soul” of the industry.

In an interview with Xconomy shortly after the op-ed was published, Holtzman said the role of CEOs should be about more than just maximizing profits. “We have a role, as leaders in a capitalist society, to speak [about] social justice—which I would argue is a necessary condition of the flourishing of a capitalist society.” Levin added: “How do we move this country forward? The only way is for us to stand up, and to say we’re here, we have a voice, and we have things to say.”

They went on to say that for the life science industry, “speaking up” also means charging responsible prices for drugs. “A responsible industry doesn’t consistently raise prices every year without increasing benefit,” said Levin.

Levin told Xconomy in an email last week that he has been speaking one-on-one with U.S. senators and members of Congress, but hasn’t made much headway. Still, he says, “I see much more willingness to stand up from my biotechnology colleagues” and vows to keep speaking out.

Institute for Clinical and Economic Review – Watchdog
For several years, ICER has been issuing reports on the cost effectiveness of new drugs, often saying that the drugs’ benefits are not enough to justify their high prices. Over the last year, ICER’s influence has grown. A few drug makers, rather than immediately attacking ICER as others have done, have instead worked with the watchdog and taken its assessments into consideration when coming up with a price for their products. Some have even priced their new drugs within ICER’s suggested range.

Regeneron Pharmaceuticals, for instance, said in March that it would slash the price of its expensive cholesterol drug, alirocumab (Praluent), for payers that agree to work with the company. Regeneron’s decision came after the company submitted key clinical data on alirocumab to ICER before publicly releasing the data, so that it could be included in ICER’s review. In May, Regeneron said it cut a deal with the pharmacy benefit manager Express Scripts, which will reduce the price of alirocumab to the lower end of ICER’s range—a price two to three times lower than last year’s.

Another sign of ICER’s growing power was a program announced this week by CVS Health, which runs one of the country’s largest pharmacy benefit managers. CVS said it would allow clients, such as employers who buy health insurance for employees, to exclude drugs from coverage if ICER says the treatments exceed a certain cost-effectiveness threshold.

Timothy Lu – Scientific Founder
Timothy Lu has been starting life-science companies since his graduate school days, starting with Sample 6, a food diagnostics developer. He hasn’t slowed down since becoming an MIT professor in 2010. In the past five years, Lu, a synthetic biology researcher, has ramped up his startup activity, including Synlogic (NASDAQ:SYBX), which is developing engineered bacteria as therapeutics, BiomX (microbiome drug discovery), Eligo Bioscience (targeted antimicrobials), Engine Biosciences (rapid drug discovery using genomics and artificial intelligence), and Senti Biosciences.

With Senti, Lu is taking a sabbatical from MIT to be the company’s founding CEO. The San Francisco-based company is using synthetic biology to engineer human cells with new gene circuits to give them therapeutic properties. The company says its technology could be used to make cell and gene therapies, such as CAR-T cell therapies for cancer, more precise and with fewer side effects. It’s targeting cancer and autoimmune diseases, and announced earlier this year a $53 million round of investment.

Feng Zhang – Discoverer
The Broad Institute’s Feng Zhang is best known for being a key developer of the CRISPR-Cas9 gene editing system. But he and his lab have kept on discovering ways to improve and expand the use of genome editing. In 2015, his lab reported a new version of CRISPR that uses a different DNA-cutting enzyme than Cas9. Known as Cpf1 or Cas12a, it cuts DNA differently than Cas9 and might offer a better way to insert a piece of DNA.

In 2017, Zhang’s group detailed another CRISPR system that edits RNA rather than DNA, and could end up treating certain diseases without permanently altering the genome. The RNA-editing technology is now part of Beam Therapeutics, which was co-founded by Zhang and other CRISPR pioneers (including David Liu of the Broad Institute, a finalist in the Big Idea category).

In 2018, scientists from Arbor Biotechnologies, which Zhang co-founded, published a paper describing another CRISPR Cas enzyme, Cas13d, which cuts RNA. It is much smaller than other RNA-snipping enzymes and could potentially be easier to deliver into human cells as a therapeutic.

Other members of the growing cast of Cas enzymes made yet another appearance this year, in the form of a diagnostic. Zhang’s SHERLOCK system, featuring Cas12a and Cas13 enzymes, can detect the presence of RNA from viruses such as the Zika virus and DNA from tumors.