Targeted in vivo delivery of genetic medicines utilizing an engineered lentiviral vector platform results in CAR T and NK cell generation.

ABSTRACT: The development of chimeric antigen receptor (CAR) T cell therapies has greatly impacted the treatment of B cell malignancies; however, manufacturing these patient-specific, autologous cell therapies is complex, costly, and requires preconditioning chemotherapy prior to infusion, limiting patient access. Here we describe the development of a lentiviral platform based on a novel, detargeted viral fusogen (Gen 2.1 Fusogen) and a membrane-bound targeting moiety to enable in vivo targeted delivery of stably integrating genetic medicines without the need for lymphodepletion. INT2104 employs a single chain variable fragment (scFv) targeting CD7 ("CD7 Binder") to deliver a CAR20 transgene to CD7⁺ T and natural killer (NK) cells. Preclinical data generated in mouse and cynomolgus macaqu

SUBMITTER: Andorko JI 

PROVIDER: S-EPMC12848171 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature