ssDNA-tLNP CAR T

ssDNA-tLNP CAR T is an in vivo cell therapy platform developed by CPTx GmbH that utilizes immune-silent single-stranded DNA (ssDNA) encapsulated within targeted lipid nanoparticles (tLNPs) to generate functional CAR T cells directly within the patient's body. This approach involves systemic administration, where the tLNPs exhibit pronounced tropism for T lymphocytes in the spleen and lymphoid tissues. Once internalized, the ssDNA serves as a versatile genetic vector, functioning either as a donor template for genomic modification or as a durable episomal expression vector, enabling sustained CAR expression without the need for viral vectors or complex ex vivo manufacturing. Preclinical studies have demonstrated that this platform enables efficient in vivo generation of CAR-expressing T cells that undergo robust expansion and achieve complete eradication of established tumors with a favorable safety profile and minimal innate immune activation.

Other names

ssDNA-tLNP encoding a CAR transgene