Research Focus Area
In Vivo Engineering: Cell Therapy Without The Cells
How it works
Cell therapies like CAR-T work by taking a patient’s T cells out of the body, engineering them in a lab, and infusing them back in. It’s powerful, but slow and expensive, because every patient’s cells have to be individually collected, modified, and grown before treatment can begin.
In vivo engineering skips that step. Instead of editing cells outside the body, it delivers the same instructions — CAR constructs, TCRs, reprogramming factors — directly into the patient, using vectors designed to find and modify specific cell types where they already live. The leading approach today uses lipid nanoparticles (LNPs) to carry mRNA to target cells, producing a therapeutic protein for a limited window before it fades — though viral and non-viral vectors capable of longer-lasting changes are also being explored.
Why it matters
Cell therapy's biggest barrier isn't always the science — it's the logistics. Manufacturing a single batch of CAR-T cells can take weeks, costs hundreds of thousands of dollars, and has to be repeated for every individual patient. In vivo engineering points toward something fundamentally different: a product manufactured once, at scale, and administered the way a normal infusion or injection would be.
This is still early. The technology has only recently emerged in a handful of labs, and most of the foundational questions — how to target the right cells, how long an effect should last, how to measure where the therapy actually goes in the body — remain open. But the potential payoff is significant: an off-the-shelf alternative to cell therapy, built to be simpler and more accessible from the start rather than retrofitted to be so later.
What we’re doing
- Comparing stable versus transient gene expression to understand which approach fits which clinical setting, and whether repeat dosing is a real obstacle or simply a different tradeoff than today’s one-and-done cell therapies.
- Investigating how to target vectors, viral and non-viral, to precise cell types, including the use of antibodies, ligands, and synthetic binders to guide delivery.
- Studying biodistribution to understand where these therapies actually go in the body, and to rule out unwanted effects on healthy tissue.
What’s next
- Determining whether DNA-based approaches can offer a more stable, longer-lasting alternative to current mRNA platforms.
- Applying this technology to produce other therapeutic tools directly in the body, including antibodies and cell engagers.
- Building toward a true off-the-shelf cell therapy — that doesn’t require harvesting a single cell from the patient it’s meant to treat.