Writing a lasting gene with RNA
Its two-part treatment delivers RNA in lipid nanoparticles. One component produces a gene-insertion protein; the other carries the therapeutic gene. After insertion, the protein and RNA break down, leaving the gene at the targeted site.
Early development before clinical trials
The company reports proof of concept in humanised mouse models. Its first studies in nonhuman primates were planned for late 2026. The approach is intended to modify immune cells inside the body rather than manufacture a separate cell treatment for each patient.
Where the funding goes
- Development of the first in vivo CAR T candidate
- Further preclinical studies of the gene-insertion platform