In Vivo CAR-T
In Vivo CAR-T
In vivo CAR-T therapies deliver CAR-encoding vectors (lentivirus, AAV, LNP, etc.) directly into the patient, reprogramming T cells in situ and eliminating the need for ex vivo cell manufacturing and reinfusion. While this dramatically improves accessibility, scalability and cost, it also removes the ex vivo safety gate of fully characterizing and releasing the final cellular drug product. As a result, the burden of safety and efficacy justification shifts upstream — onto the sequence fidelity, genome integrity and purity of the vector itself, together with its integration behavior, transgene expression, and clonal dynamics over a multi-year horizon. Regulators have responded in kind: FDA Considerations for the Development of CAR T Cell Products (2024), FDA Long-Term Follow-Up After Administration of Human Gene Therapy Products (2020, with follow-up of up to 15 years for integrating vectors), ICH S12, ICH Q5A(R2)/Q5B/Q6B and NMPA Technical Guidelines for In Vivo Gene Therapy Products (2022) collectively raise the bar for in vivo CAR-T quality control and IND/BLA submission.
ZhuHai GeneRulor's In Vivo CAR-T Solution Center is built on third-generation long-read sequencing (Oxford Nanopore / PacBio) as the analytical core, complemented by short-read NGS for orthogonal verification. The platform is organized around two strategic pillars — Vector Nucleic Acid QC and Long-Term Follow-Up (LTFU) Monitoring. Upstream services cover full-length plasmid sequencing, AAV ITR integrity, lentiviral RNA integrity and splicing analysis, particle nucleic acid purity, and AAV genome integrity with ITR characterization; downstream services extend to vector integration site and proviral structure analysis, full-length transcript profiling, and single-cell / bulk immune repertoire sequencing (TCR/BCR). Longitudinal integration site analysis (ISA), clonal expansion assessment and vector persistence monitoring together provide continuous safety surveillance across the clinical and post-marketing phases. All services are delivered under GMP-aligned operations with ISO 9001 / CNAS / CAP accreditation, producing CTD- and PVP-ready data packages that support dual IND submissions to both NMPA and FDA.
