Meet the young women unlocking new ways to treat diseases and shaping the future of medicine

Luhan Yang, chief scientific officer at eGenesis, was born and raised in China, where she studied psychology and life sciences before moving to the US to get her Ph.D. at Harvard. It was there where she met George Church and started working with the human genome, and ways to modify it, especially using the CRISPR gene-editing technology.

Her work in the lab led to her and Church in 2014 founding eGenesis, a company that's using the CRISPR to make pig organs viable for transplants into people, an advancement that could impact the hundreds of thousands of people waiting for transplants around the world.

There are two huge hurdles to getting animal-organ transplants to successfully work in humans — a process known as xenotransplantation. The first is the virus, which in August the eGenesis team managed to get past when it had produced 37 piglets that had inactivated Porcine Endogenous Retrovirus, or PERV. The work set a record for the most complex edits made with CRISPR. The virus, which is part of the pigs' DNA, has been an issue for human-pig transplants in the past because of concerns that it could infect humans.

The second that Yang and eGenesis are still working on, is the immunology. Since the pig organ would be foreign to the body, the person's immune system might try to kick it out, rejecting the organ. Those proved too challenging for a slew of researchers going after this subject in the 1990s, but Yang is hopeful that her approach of building up the company's platform will help researchers get to the heart of the issue.

The contribution the under-40 generation will have on medicine: Bringing in a more global perspective to medicine. For example, Yang's background led her to work with xenotransplantation, she said, in part because of the historically limited access to donor organs Asian countries have had for transplants.

Erica Weinstein, an associate at the venture firm Flagship Pioneering, wanted to ask "why not" during her doctorate research in immunology at the Icahn School of Medicine at Mout Sinai. She was interested in taking seemingly "crazy" ideas and turning them into therapies that could be developed and one day help treat patients. So Weinstein joined Flagship in 2015 to put those ideas to the test.

"That questioning is what Flagship does," Weinstein said. There, she's helped develop the pipelines of new startups, helping them go from a concept to a team of 20 to 30 people. So far, she's cofounded Cygnal Therapeutics, which is working on treatments for cancer, immunologic diseases and regeneration, and two companies that are still in stealth mode.

The contribution the under-40 generation will have on medicine: The passion to bring life-changing therapies to reality and by being willing to think, "This feels weird, but I'm probably going down the right path," Weinstein said.

Stephanie Tagliatela, cofounder and chief scientific officer of Encoded Genomics, met Kartik Ramamoorthi, cofounder and CEO, while they were Ph.D. students in the brain and cognitive-sciences department at MIT. Ramamoorthi was also included on BI's "under 40" list. Through his work, he got the idea to start his own company that focused not just on gene therapy but gene regulation as well.

Gene regulation is the mechanism through which some cells express certain genes and others don't. So far, there's only one approved gene therapy in the US, according to the FDA. The hope is that by using gene therapy to manipulate the way certain cells express genes, you might be able to treat genetic diseases we have a hard time treating now. The ability to take the concept from idea to patients quickly convinced Tagliatela to leave her Ph.D. work early to join the company on the West Coast.

So far, Encoded has raised $50 million from investors including genetic-sequencing giant Illumina, Arch Venture, and Venrock, and plans to give more details about the diseases they're going after in 2018.

The contribution the under-40 generation will have on medicine: Tagliatela said that drug development could experience big changes thanks to genomic advancements. "The onset of big data is going to be a really exciting area for drug development that I think our generation will be really key to advance," she said.

Mary Rodgers, a senior scientist at Abbott Laboratories, has a fun way to describe her role: virus hunter. After attending the University of Madison in undergrad for biochemistry, Rodgers attended a Ph.D. program at Harvard University, where her work focused on the hepatitis virus. She then went on to do research at the University of Southern California before returning to the Midwest to work at Abbott.

Her job: Makes sure that we can detect infections no matter where they are, based on Abbott's database of viruses. That can be a big deal for diseases like HIV and hepatitis. Diagnostic tests (which Abbott makes) have to catch all different mutations to the virus so that patients don't get a false negative result. Rodgers' job is to stay ahead of that curve.

The contribution the under-40 generation will have on medicine: The ability to edit genomics with CRISPR, which could have big implications for infectious disease, Rodgers said.

Maria Pereira, chief innovation officer of Gecko Biomedical, went to the Massachusetts Institute of Technology after studying pharmaceutical sciences in Portugal. As a Ph.D. student in bioengineering, she worked on a project with the Boston Children's Hospital, working on better ways to repair and regenerate hearts, particularly in babies. The problem wasn't easy to solve, but in the end it led Pereira to develop a material the consistency of honeythat when a certain light's applied turns into a bendable, dissolvable structure.

"I always liked to create," Pereira said. From there, she was able to turn her project into a company. The platform of polymers she created have the potential to work as a sealant, as a 3D structure that acts like scaffolding to help tissue grow around it, or as a way to deliver drugs that slowly release as the material breaks down. Because the substances are biodegradable, allowing the body to heal on its own, it could transform certain surgical procedures that currently leave lasting implants in your body.

The contribution the under-40 generation will have on medicine: The younger generation of leaders is used to moving fast and has been exposed to technology starting at a young age. The under-40 generation, she said, will have a chance to "go beyond" and bring different areas of science and technology together.

Julia Oh, assistant professor at the Jackson Laboratory, had two main loves coming out of her Ph.D. work at Stanford: microbes and synthetic biology, which is the reprogramming of cells to have them do what you want them to do.

Now, through her work at Jackson Labs, a nonprofit biomedical research institution, she's able to combine the two concepts to develop treatments that use engineered microbes to treat diseases. For example, say you had a skin condition (Oh's work is focused on the skin microbiome), instead of treating it with a cream, you could just introduce a microbe to the environment that would live on your skin with all the other microbes you already have. If it sensed that your skin was acting up, it could secrete a chemical that would counteract it. The potential for engineered microbes is broad, Oh said, ranging from autoimmune disorders, to cancer, to bacterial infections.

The contribution the under-40 generation will have on medicine: Oh said it would be embedding technology into biology, including having biologists build that technology themselves. It'll also be the willingness to collaborate with people who are experts in areas the researcher might not know much about, such as physics. "We don't even know the questions they can answer," Oh said.

Rebecca Leary, a senior investigator at Novartis Institutes for Biomedical Research, is working on blood tests to help researchers better understand how a tumor's responding to a particular experimental treatment during clinical trials.

These tests, known as liquid biopsies, look for something called "circulating tumor DNA," or the bits of DNA that are released from dying tumor cells into the bloodstream. It's a less invasive way to get a snapshot of a tumor than a traditional tissue biopsy. The liquid biopsies Leary works with get a comprehensive look at mutations in tumor DNA, especially keeping an eye out for mutations we don't know a lot about just yet.

Leary received her Ph.D. from Johns Hopkins University, where she was working with cancer genetics, trying to find new cancer targets, before joining Novartis.

The contribution the under-40 generation will have on medicine: Bringing innovative tools to patients, like those using gene editing, and finding new ways of thinking through medical challenges, Leary said.

Cigall Kadoch, a professor of pediatric oncology at the Dana-Farber Cancer Institute and Harvard Medical School and cofounder of Foghorn Therapeutics, was in college around the time the first human genome was sequenced, a moment that would shape her career. After the entire genome was sequenced, researchers started looking into the genomes of certain diseases, including cancer.

Through her Ph.D. work at Stanford, she explored a protein complex that seems to be implicated in all of the cases of a type of sarcoma. And by changing features of the protein complex, the cancer could essentially be "reversed," an area of medicine called epigenetics.

Unlike existing drugs, the idea here is to reverse the effects of cancer by shutting off the genes responsible for cancer growth. The genes that'll be shut off are "master gene controllers," Kadoch said, meaning they're responsible for what's causing to grow aggressively or spread around the body. In 2016, Kadoch cofounded Foghorn Therapeutics as a way to turn her work in reversing the effects of mutated genes into treatments.

The contribution the under-40 generation will have on medicine: Translating those gene mutations that we understand are connected to cancer into actionable therapeutic targets that can succeed in curing cancer.

While doing her Ph.D. at the University of California at Berkeley in 2007, Rachel Haurwitz, CEO of Caribou Biosciences, had the "luck" to work with an exciting new technology called CRISPR. At the time, the revolutionary gene-editing took only had one paper that had been written about it.

The more she learned, the more she realized how much of the technology had the potential to be used not only in academic research but also in everything from therapeutics, to agriculture, to livestock. Haurwitz, along with a team of scientific cofounders including biochemist Jennifer Doudna, a professor at UC Berkeley who was instrumental in early work on CRISPR, created Caribou as a platform tech company.

Through Caribou, partner companies like Novartis and DuPont are able to license CRISPR for use in their products. In addition to external partnerships, Caribou's plan is to also get into some commercial markets as well, or potentially spin out companies, as the company did with Intellia Therapeutics, a company exploring how to apply CRISPR to medicine.

The contribution the under-40 generation will have on medicine: This generation will be willing to take big bets and be way more comfortable combining science and technology, pushing the field forward, Haurwitz said.

Arpa Garay recently became the new vice president of vaccines at Merck after spending two years working in Norway as a managing director. During the 10 years she has spent at Merck, she's worked in a range of disease areas from diabetes to cancer.

Now as the head of vaccines, she's overseeing the area during a time when the business model of pharmaceutical companies is experiencing a big change. Garay is tasked with growing the vaccine business, which includes vaccines for human papilomavirus, pneumonia, and chicken pox, among others.

The contribution the under-40 generation will have on medicine: It'll be by using data better, more efficiently, and having a more global mind-set, Garay said.

Kyla Driscoll, a senior research adviser and group leader at Lilly, is working to develop new cancer immunotherapy treatments that target TGF-beta, a cytokine (proteins that are important in cell signaling) that suppresses the immune system in seriously hard-to-treat cancers including glioblastoma and pancreatic cancer. The hope is that by finding new ways to target the immune system, more people with these tough-to-treat cancers might be able to use their own bodies to fight cancer cells.

Driscoll got her Ph.D. from Rutgers University, where she studied how viruses can induce cancer and working with cancer vaccines.

The contribution the under-40 generation will have on medicine: More clinical trials that are more "judicious" about finding people who will respond the best, Driscoll said.

Colleen Cuffaro, a principal at Canaan Partners, started her career studying chemistry at the University of Pennsylvania before getting her Ph.D. in cellular and molecular physiology at Yale. During that time, she was introduced to investing and venture capital.

Cuffaro joined Canaan in 2014 and has since led the $38 million investment round for Arrakis Therapeutics, a company that's working on RNA-targeted drugs. She's also the CEO of a small biotech that's still in stealth mode.

The contribution the under-40 generation will have on medicine: It'll be on focusing on genes, rather than proteins, which is how most drugs work today. "Building on those advances and integrating the chemistry, biology, and computational knowledge that exists today, our generation is thinking outside the (protein) box, if you will," Cuffaro said. "Shifting the focus to modulating genes brings us to entirely different modalities — gene therapy, gene editing, cell therapies, drugging RNA."

Viki Bockstal, the biomarker lead for filovirus vaccines at Johnson & Johnson's Janssen Vaccines, helps develop vaccines to prevent hard-to-treat conditions, including HIV, polio, and Ebola. She joined Janssen in 2012 after receiving her Ph.D. in bioengineering sciences at Vrije Universiteit Brussel.

Her job is to help Janssen's Ebola vaccine keep moving through clinical trials, a process that can be difficult when there isn't an Ebola outbreak. In the absence of that, Bockstal determines the parameters that help determine whether the vaccine is effective or not.

The contribution the under-40 generation will have on medicine: Bockstal said it will be by finding vaccines or cures for some of the infectious diseases and conditions that have so far evaded our grasp, such as Ebola, HIV and cancer. "I truly believe this generation has the best shot at not only making this happen, but also really eradicating diseases such as AIDS, measles, polio, tuberculosis, and malaria," Bockstal said.

Stephanie Barrett, a principal scientist at Merck, is working to make implantable devices to treat infectious diseases. Originally from Canada, Barrett moved to the US in 2004 to get her Ph.D. in chemistry at the University of North Carolina at Chapel Hill. Shortly after completing her graduate work, she joined Merck.

Right now, treatments for diseases such as HIV and hepatitis are taken via pills. Merck's hope is that by building an implanted device, people will do better on the medications because they will actually adhere to them. Adherence is key when you're thinking about infectious diseases like HIV where you need low viral counts.

The contribution the under-40 generation will have on medicine: The energy and "hungriness" this generation will have will propel things forward, Barrett said.

Narges Bani Asadi, the vice president and Life Cycle Leader of sequencing genomics at Roche Sequencing Informatics, grew up in Iran before moving to Stanford University for graduate school. While pursuing a master's and later a Ph.D. in electrical engineering, she met a mentor who explained to her a future in which biology and medicine is more about data than a biology or chemistry lab problem. "It blew my mind," she said.

From there, she started working at that intersection between computer science and medicine, launching a startup called Bina that was later acquired by Roche. The company worked to find clinical applications for genomic data, so that the findings that come out of academic researchers make it to patients.

"How can that actually change medicine? That's the bottleneck today," she said. "What is the clinical utility of this information?"

The contribution the under-40 generation will have on medicine: It will be getting more nontraditional, and possibly even younger people into the industry that come from different backgrounds and expertise areas, Bani Asadi said.