- CEO Laura Shawver
- Total raised $340 million
- Headquarters San Diego, CA
Turning a patient’s own cells into powerful disease combatants — without the complexity of removing them from the body to do so — was a fantasy. Now it’s getting closer to reality.
If drug developers like Capstan Therapeutics can make safe and effective in vivo CAR-T cell therapies, it could ease the huge scientific, manufacturing and cost challenges that have prevented transformative blood cancer treatments from being widely available. And it could open up the use of in vivo CAR-T cell therapies as a potential treatment for autoimmune diseases.
To do so, Capstan is employing the mRNA technology behind the blockbuster Covid-19 vaccines. But the work is still in the early stages, and much remains unknown.
“Two years ago, when I joined, it was kind of science fiction-y,” Capstan CEO Laura Shawver said. “Today we have line of sight [into the clinic], such that we’re going to understand, can this work in people and really be transformative?”
Founded in 2021 as Teefib Biosciences, Capstan is bridging the worlds of its esteemed scientific founders from the University of Pennsylvania, including Nobel laureate Drew Weissman, CAR-T cell therapy pioneer Carl June, and cardiologist and stem cell biologist Jonathan Epstein. The startup’s approach draws from a 2022 Science paper published by the Penn researchers showing that a targeted lipid nanoparticle could be used to deliver mRNA to T cells and reprogram them into CAR-T cells — powerful agents of the immune system.
Since its founding, It’s raised a total of $340 million in venture backing, from big names including Forbion, Sofinnova, RA Capital, Polaris Partners and others, as well as from drugmakers or their venture arms at Bristol Myers Squibb, Johnson & Johnson, Eli Lilly, Bayer, Pfizer and Novartis.
This year, regulatory agencies in Australia and the US OK’ed the first clinical tests of in vivo CAR-T therapy in cancer patients. Interius BioTherapeutics and Umoja Biopharma were given the go-ahead to run human studies of their in vivo CAR-T therapies in the two countries, respectively.
However, both companies are developing in vivo CAR-T treatments using an older technology with lentiviral vectors, as opposed to mRNA delivered via a fatty acid bubble. Moderna has also invested in in vivo cell therapy using mRNA. But it’s not yet known which approach — viral or nonviral — will yield the best results.
While traditional CAR-T therapies have made their name as cancer treatments, recent clinical data have suggested they could be dramatically effective treatments for autoimmune disease as well. Capstan says it plans to start its first human studies in autoimmune disorders, though Shawver declined to lay out when the company expects to begin clinical trials.
Shawver said that the company was in preclinical development and working through manufacturing steps, noting that their therapy involves a lot of different components.
“A number of the companies are testing different technologies, and we will five years from now understand which of these technologies have entered the clinic, or will enter the clinic soon, is the more or most optimal of the in vivo approaches,” Shawver said.
Key backers: Bristol Myers Squibb, Forbion, Johnson & Johnson, Mubadala Capital, Perceptive Advisors, Sofinnova Investments, Alexandria Venture Investments, Eli Lilly, Leaps by Bayer, Pfizer Ventures, Polaris Partners, Novartis Venture Fund, OrbiMed, RA Capital Management, Vida Ventures
Find the full list of 2024 Endpoints 11 winners here.