Azalea Therapeutics Reports First-In-Primate In Vivo CAR T Data, Advancing Toward Clinical Translation

New updates have been reported about Azalea Therapeutics.

Azalea Therapeutics, Inc. has reported first-in-primate results from its in vivo CAR T cell platform, with data accepted as a late-breaking oral presentation at the 2026 American Society of Gene and Cell Therapy meeting in Boston. The study demonstrated genomic site-specific engineering of TRAC-CAR T cells in immune-competent non-human primates using a single intravenous dose, positioning the company’s technology as a potential alternative to complex ex vivo CAR T manufacturing.

Azalea’s dual-vector system combines CD3-targeted enveloped delivery vehicles carrying transient Cas9 complexes with a T cell-tropic AAV that delivers a promoterless CAR cassette flanked by TRAC homology arms, enabling precise CAR insertion at the TRAC locus under the native T cell promoter. In six rhesus macaques treated without lymphodepletion, TRAC-CAR T cells reached up to 41% of circulating T cells by Day 11 and produced greater than 90% B cell depletion in blood by Day 10, with similarly deep B cell clearance in lymph nodes and bone marrow at higher doses.

The treatment was generally well tolerated, with no deaths, no neurotoxicity, and molecular analyses showing no off-target CAR expression or integration outside T cells in blood, supporting a favorable safety profile for further development. Chief executive officer Jenny Hamilton stated that these findings mark a major milestone for Azalea and provide translational support for in vivo cell engineering aimed at safer, durable, and more physiologically regulated therapies.

Scientific co-founder Connor Tsuchida emphasized that this appears to be the first demonstration of site-specific gene integration in T cells in a non-human primate in vivo, validating Azalea’s strategy of cell-selective delivery and locus-specific editing in a clinically relevant model. The company views this dataset as a key step toward human trials of in vivo CAR T therapies across oncology, autoimmune disease, and genetic disorders, with the potential to expand access and reduce manufacturing costs compared with conventional autologous CAR T approaches.