Pharmas back a startup’s plan to make CAR-T cells inside the body

Biotech
Wednesday, September 14th, 2022 9:04 am EDT

Dive Brief:

  • Biotechnology startup Capstan Therapeutics said Wednesday it raised $102 million in Series A funding, gaining support from pharmaceutical and venture capital firms to fuse messenger RNA and CAR-T technologies together in hopes of engineering therapeutic immune cells within the body.
  • CAR-T innovators Carl June and Bruce Levine, and mRNA pioneers Drew Weissman and Hamideh Parhiz are among the scientific founders of Capstan, which got off the ground in November 2021 with $63 million in seed funding.
  • The company plans to use the type of mRNA technology that underlies the COVID-19 vaccines from Pfizer and Moderna to re-engineer disease-fighting T cells into CAR-T cells. The Food and Drug Administration has approved six CAR-T therapies for several blood cancers, but they are laborious to make and expensive.

Dive Insight:

CAR-T therapies are a scientific and clinical advance, but they remain complex products to make and use. They’re constructed from the T cells of individual patients, which are drawn, re-engineered, manufactured and then re-infused into patients, a process that can take weeks and requires patients to stay near a hospital while their treatment is prepared.

Drugmakers have tried to overcome these limitations by either using donor cells as an “off-the-shelf” product — potentially risking rejection by the body — or by manipulating cells directly inside the body. Companies like Allogene Therapeutics, Precision BioSciences and Caribou Biosciences have been working on off-the-shelf CAR-T therapies, while Ensoma, Vector BioPharma, Umoja Biopharma and Interius BioTherapeutics aim to re-engineer T cells and other disease fighting cells in the body, as Capstan is planning.

Capstan’s vision relies on the approach used to great success in the mRNA vaccines to turn T cells into targeted therapies. Encapsulated inside a lipid nanoparticle, or a small fatty sphere, Capstan’s medicines would be designed to deliver an mRNA payload that redirects a patient’s T cells toward diseased cells.

The company’s founders have so far used the technology to target fibrotic heart cells in diseased mice.

Capstan said its goals are to develop therapies for conditions for which there are no effective treatments, as well as therapies for certain inherited blood disorders.

The Series A round announced Wednesday was led by Pfizer with funding from Bayer, Eli Lilly, Bristol Myers Squibb, Polaris Partners, Alexandria Venture Investments, as well as all previous investors. Those include the seed round investors Novartis, OrbiMed, RA Capital, and Vida Ventures.

The company will be run by CEO Laura Shawver, who previously headed Synthorx until its $2.5 billion acquisition by Sanofi in 2019, as well as Silverback Therapeutics, which was bought earlier this year by ARS Pharmaceuticals for its market listing after Silverback’s own cancer research failed.

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