
Biotechnology Developer
▲October 26-27, ChengduNote:This article does not constitute any investment opinions or recommendations., subject to official/company announcements;This article only introduces drugs related to medical health, not a recommendation of treatment options (if involved), and does not represent the platform's position.Any article reprinted must be authorized.
Turning patients' own cells into powerful disease fighters — without removing them from the body — is a fantasy. Now it is getting closer to reality.
If like Capstan TherapeuticsIf such a drug developer can create a safe and effective in vivo CAR-T cell therapy, it could alleviate the significant scientific, manufacturing, and cost challenges that have hindered the widespread use of this transformative blood cancer treatment. It could also pave the way for in vivo CAR-T cell therapy as a potential treatment for autoimmune diseases.
To this end, Capstan Therapeutics is adopting the mRNA technology behind the groundbreaking Covid-19 vaccine. However, this work is still in its early stages, and there are many unknowns.
"Two years ago, when I joined, it felt a bit like science fiction," said Laura Shawver, CEO of Capstan Therapeutics. "Today, we have [a clear path to the clinic], so we’ll soon understand whether this can work in humans and truly be transformative."
Capstan Therapeutics, Inc., founded in 2021 as a spin-off from Teefib Biosciences, serves as a bridge for 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 is based on a scientific paper published in 2022 by researchers at the University of Pennsylvania, which demonstrated that targeted lipid nanoparticles can be used to deliver mRNA to T cells and reprogram them into CAR-T cells — powerful agents of the immune system.
Since its establishment, it has raised a total of $340 million in venture capital support from well-known companies including Forbion, Sofinnova, RA Capital, Polaris Partners, as well as from the venture capital arms of pharmaceutical manufacturers such as Bristol Myers Squibb, Johnson & Johnson, Eli Lilly, Bayer, Pfizer, and Novartis.
This year, regulatory agencies in Australia and the United States approved the first clinical trials of CAR-T therapy administered directly in cancer patients. Interius BioTherapeutics and Umoja Biopharma have been authorized to conduct human studies of their in vivo CAR-T therapies in these two countries.
However, both companies are developing in vivo CAR-T therapies using older lentiviral vector technology rather than mRNA delivered via fatty acid bubbles. Moderna has also invested in in vivo cell therapies using mRNA. But it remains unclear which approach, viral or non-viral, will yield the best results.
Although traditional CAR-T therapies are well-known for cancer treatment, recent clinical data suggest that they may also be a highly effective approach for treating autoimmune diseases. Capstan Therapeutics stated that it plans to initiate the first human study on autoimmune diseases, but Shawver declined to disclose when the company expects to begin clinical trials.
Shawver said that the company is in the preclinical development stage and completing production steps, noting that their therapy involves many different components.
Shawver said, "Many companies are testing different technologies, and in five years we will know which of these have entered or are about to enter clinical trials, representing more or the most ideal in vivo approaches.
Main Supporters: Bristol-Myers Squibb, Forbion, Johnson & Johnson, Mubadala Capital, Perceptive Advisors, Sofinnova Investments, Alexandria Venture Investments, Eli Lilly and Company, Leaps by Bayer, Pfizer Ventures, Polaris Partners, Novartis Venture Fund, OrbiMed, RA Capital Management, Vida Ventures.

Source: Capstan Therapeutics
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[About Capstan Therapeutics]
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