Nvelop launches to deliver gene-editing cargo

Nvelop Therapeutics has launched to improve gene therapy delivery. The company was first founded in 2022 with $100 million in funding from investors on the basis of independently developed platforms advanced by David Liu at the Broad Institute of MIT and Harvard and Keith Joung of Massachusetts General Hospital.

The company is developing viruslike delivery mechanisms for gene-editing cargo. Nvelop says its delivery agents can enable gene editing without taking cells out of the body, editing them, and putting them back in. This advance can make the technology applicable to more conditions, the company says.

“There [are] so many gene modifications that can be accomplished,” says Melissa Bonner, Nvelop’s chief scientific officer. “But they can only effectively be accomplished either ex vivo—outside the body—or in very limited target tissues.” That’s because delivery systems aren’t yet able to deliver to broad tissue types, she says.

First reported in the journal Cell in 2022 (DOI: 10.1016/j.cell.2021.12.021), the platform from Liu and the Broad Institute involves engineered viruslike particles that can deliver proteins and RNA complexes necessary for Cas9 gene editing and base editing. In 2023, Liu’s team reported that it could achieve prime editing using the same delivery mechanisms (Nat. Biotechnol. DOI: 10.1038/s41587-023-02078-y).

Joung’s platform is similar to Liu’s, but it uses fewer viral components. And while the Liu platform uses Gag proteins, which are derived from viruses, the Joung platform does not.

And it is possible that the Joung platform could use human-derived envelope proteins instead of viral envelope proteins on the delivery particle surface. This feature could lead the host immune system to ignore these particles, which could minimize adverse reactions.

While Nvelop did not provide specifics, it says the flexibility of these platforms will allow it to deliver gene-editing machinery to specific tissues.

Nvelop is developing the two platforms simultaneously, Bonner says, and may be able to transfer characteristics from one to the other. For example, it may be possible to use Joung’s human-derived envelope proteins with the Liu platform.

Jeff Walsh, the CEO of the company, says Nvelop can produce its own gene-editing cargo or partner to provide the delivery mechanism for another company’s gene-editing therapy. However, Nvelop has not established any concrete partnerships, nor is it ready to disclose any conditions it hopes to treat, he says.