Yael Weiss, MD, PhD, is no stranger to rare disease research. Weiss, the founder and CEO of rare disease biotech Mahzi Therapeutics, started her career as the medical director of Genzyme, a rare disease biotech famous for creating a treatment for Gaucher disease before being acquired by Sanofi in 2011.
At that point, Weiss had been in Big Pharma for eight years herself, as medical director, then director of global scientific affairs and executive director of regional deals at Merck. Still, Weiss would eventually find herself drawn back to rare disease research.
“I learned that the smaller the company, the more comfortable I feel,” Weiss said.
In an exclusive interview with Cure, Weiss shared her journey as a female founder and entrepreneur in the rare disease space, and tips for other women in life sciences leadership.
From Academia to Industry: A Path to Bringing Science to the Clinic
Weiss completed her undergraduate, graduate and medical school training in Israel before moving to the United States. It was in medical school at the Hebrew University of Jerusalem where she realized her path lay in bringing science to the clinic.
“That's where I really realized that my passion lies in the interface between medicine and science and the translational part of it,” she said, “taking science and translating it into good medicines.”
She felt that working in the biotech and pharma industry would be the best place to make an impact at this critical intersection. Working in industry, Weiss said, is the best place “to mentor scientists and academics on how to orient their research towards a more practical aspect,” like turning basic lab discoveries into new drugs.
Rare genetic diseases were also always a passion, Weiss said. She completed her PhD research at the Weizmann Institute of Science in Israel in Down syndrome, and worked at rare disease companies Genzyme and Ultragenyx before founding San Francisco-based Mahzi Therapeutics in 2021.
At Ultragenyx, Weiss said, “I had a chance to interact with a lot of parents and patient advocacy groups, which led me to the idea of starting Mahzi to continue supporting these groups by developing therapies with them.”
That’s what sets Mahzi apart from other rare disease biotech companies: it works closely with patient advocates to advance therapeutics through clinical trials and, hopefully one day, into the marketplace.
Tips for Female Founders in Biotech
The year before Weiss founded Mahzi, fewer than 10 percent of pharma and biotech CEOs were women, according to a BioPharmaDive analysis. In the nearly five years since, not much has changed.
Weiss, who has been in the pharma and biotech industry for more than 25 years, said that by now she is used to being in a male dominated field.
“Throughout my career, I worked in places that were very heavily male dominated,” she said, “I always took it as a given.”
For other women who are interested in launching a biotech company, Weiss had some tips. She suggested looking for women-led efforts, whether those are venture capital groups, other biotech companies or firms that prioritize employing women.
“Find a group that understands you better and can support you and your journey,” she said.
One group that Weiss herself belongs to is the Biotech CEO Sisterhood, a group of approximately 400 members of female biotech CEOs that uplift and advise each other. Weiss said that in a world where venture capital is still heavily male dominated, it is helpful for women to have a place for feedback, guidance and pep talks.
She also recommends the Termeer Foundation, a nonprofit organization that empowers healthcare innovators. While not exclusively focused on women, the Termeer Foundation provides fellowship programs for first time CEOs of biotech and life sciences companies.
Weiss also pointed out that in the rare disease space, most of the patient advocates that she interacts with are mothers. She said that it has become a very collaborative environment, but that some people mistakenly underestimate them.
“A lot of people say, ‘oh, these are just mothers sitting around a kitchen table,’” Weiss said. “Most of them are extremely powerful and strong women and they are the most motivated people I've ever worked with.”