As adults, few of us are doing what we wanted to grow up to be as children. Reality and bills and basic human limitations persuade us away from our cowboy ninja and time-traveling detective dreams. Fortunately, this isn’t the case for Helen Sabzevari. “I think the very first time I recognized I wanted to do what I’m doing currently, was when I was 12 years old,” she says, recalling to Forbes how she was mesmerized by a pre-teen biology lesson on cancer. 

Forty-seven years later, Sabzevari is CEO of Precigen, a biopharmaceutical company developing cutting edge treatments for a number of different diseases, including type 1 diabetes and certain forms of cancer. It’s here that Sabzevari, 59, is bringing her lifetime of experience in cancer research to realize a vision of immunotherapies that are both more effective and easier to scale than what’s on the market now.    

One of the biggest innovations in cancer treatments was the development of CAR-T cell therapy. These engineered immune cells are delivered to the body through specially designed viruses, where they target molecular changes typically seen in cancers and attack. The first such treatment was approved by the FDA in 2017, and four others have been approved since. 

CAR-T therapies have shown great success against certain cancers but they also have limitations: they don’t work well against solid tumors, and so are generally used against leukemia and other blood cancers. They’re also expensive: a course of CAR-T therapy can cost over four hundred thousand dollars, according to the Centers for Medicare and Medicaid Services.

Sabzevari’s vision is a different approach: developing what her company calls “Ultra CAR-T.” In this therapy, T-cells fighting cancer within a patient are isolated, and the genes triggering their production are introduced into plasmid DNA, a type of DNA commonly found in bacteria. The plasmid DNA is then engineered to produce T-cells that are even more potent against cancer cells. The results are then reintroduced to the body along with an IL-15 cytokine membrane, where they go to work fighting cancer. By using plasmids instead of viruses, Ultra CAR-T cells avoid the risk that the body may mistake the treatment for an invading cell that needs to be fought off, improving safety. 

Additionally, plasmid DNA can be scaled and manufactured much more cheaply and Precigen uses a sophisticated, proprietary system to ensure quality control—its manufacturing system received FDA clearance in fall 2020. It’s also fast: if a patient has their blood drawn in the morning, the T-cells can be isolated and Ultra CAR-T cells manufactured and ready to deliver back the next day, Sabzevari says. 

Another advantage of Ultra CAR-T cells is persistence. Whereas CAR-T cells only stick around the body for 2-3 weeks, preliminary data suggests Ultra CAR-T cells can stick around for months, meaning that they can attack solid tumors. This broadens the scope of cancers that could be treated with the therapy. And if it turns out there’s something going wrong with the treatment, it comes packaged with a genetic “kill switch” that gets rid of the cells.

The company’s first two Ultra CAR-T treatments are currently in phase one clinical trials, but preliminary data have so far been positive. This has made the therapy attractive not only to patients but also Wall Street. The company’s treatment approach has the potential to “disrupt current approaches with lower cost of goods, next-day delivery, and extension of efficacy into solid tumors,” Wells Fargo analysts wrote in a recent research note. 

And the woman behind that disruption? She’s been preparing for this her whole life. 

Born in Iran, Helen Sabzevari excelled in her studies, especially when it came to science and biology. Due to the country’s revolution in 1979, her family sent her to live in the United States. Thanks to her advanced work in high school in her home country, she was able to enroll in City University of New York right away. While she was pursuing her undergraduate and graduate studies, she volunteered at Sloan Kettering and the Brookhaven National Laboratory. 

After getting her PhD in cell and molecular immunology at the Scripps Research Institute, she remained at that institution to pursue her postdoctoral studies in cancer immunotherapy, where she explored the intersection of the immune system and cancer — an area where researchers are ubiquitous today but not so much back then. “I was one of the very, very few postdocs in the field at that time,” she recalls.  

She went on to do a second postdoc at Scripps exploring autoimmunity; then, in 1998, she joined the National Institutes of Health, leading an immunology group at National Cancer Institute. Her team focused on the design and development of new types of cancer treatments, include vaccines and immunotherapies. In that role, she says, “we were able to learn how to translate what we were developing in research for the patients in the clinic.”  

A job at Merck as a senior VP in immuno-oncology and global head of immunotherapy followed, in 2008. “That was a very difficult decision,” she says of choosing to leave the NCI. But as important as she felt her work at NCI was, it didn’t involve creating the drugs that would actually get to patients. “I really wanted to learn that part and bring it back to be a bridge between academia and pharma,” she says. 

After six years at Merck, Sabzevari cofounded and became chief science officer of Compass Therapeutics, a Cambridge, MA-based biotechnology startup developing antibody therapies for cancer. The move to biotechnology startups from big pharma, she says, was to get closer to the action. “I wanted to move to a smaller biotech on the cutting edge of what I considered the next innovative area in immuno-oncology,” she says. “Which was gene and cell therapy.”

She spent three years at Compass Therapeutics before signing on to Precigen as head of R&D in 2017, and became president a few months later. At the time, Precigen was a subsidiary of Intrexon Corporation but in January 2020, Intrexon merged with the Precigen subsidiary, changed its name and Sabzevari took the reins as president and CEO. Since then, the company has not only been pursuing cancer therapies, but also autoimmune disorders such as Type 1 diabetes. 

To engage type 1 diabetes specifically, the company has developed a platform where genetically-modified gut bacteria are introduced to a patient. The bacteria then produce immune cells that prevent the immune system from attacking its own pancreas. Phase one trial data for that treatment is also positive so far, and trials are moving on to phase two. The company is using the same bacteria platform to tackle other autoimmune diseases like Celiac disease. 

In Sabzevari’s first year as CEO, the company saw $103 million in revenue, a 14% increase over 2019. A little over $20 million of that came from licensing and collaboration agreements, which is typical for an early-stage biotechnology company. The rest of the company’s revenue comes from two subsidiaries that remained after the change from Intrexon to Precigen: Trans Ova, which provides genetic analysis for animal husbandry, and Exemplar, which produces genetically modified animal models for medical research. 

Moving forward this year, the company plans to continue advancing its pipeline of cancer and autoimmune treatments, with more clinical data expected to be reported in the late fall, though it’s still a few years away from getting products to market.  For her part, Sabzsevari is elated with the positive results she’s seen so far. Not for the success it brings to the company, but because of the hope it brings to patients. The way she sees it, “failure is very difficult to take because it means failure, basically, in extending somebody’s life.” That viewpoint is what drives her to keep her company’s work moving forward.

“The moment that this becomes a business to me and it’s not personal, that’s the moment I step down,” she says. “Because you can only push with the speed that we are pushing when you take it personally. For me, this has been personal all my life.”