On May 4, 2026 CPTx, a company developing next-generation non-viral in vivo CAR T therapies, reported that it will deliver an oral presentation showing in vivo proof-of-principle of its immune-quiet, single-stranded DNA-based delivery platform, as well as present two poster presentations at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, taking place in Boston, MA, from May 11-15.
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The oral presentation, by Head of Strategy and R&D Matthias Bozza, will include early readouts from preclinical testing of the company’s proprietary immune-quiet DNA + targeted lipid nanoparticle (tLNP) delivery system, which comprises a fully non-viral, systemically administered platform for in vivo generation of chimeric antigen receptor (CAR) T cells.
The therapeutic relevance of this approach was evaluated in a mouse model. Upon systemic administration, tLNPs loaded with CAR-encoding DNA achieved more durable tumor control relative to mRNA-based particles, consistent with the expectation that DNA-based payloads can provide a more sustained and controllable expression profile compared to transient mRNA-driven transgene expression.
"These data being presented at ASGCT (Free ASGCT Whitepaper) demonstrate strong progress towards efficient in vivo CAR T engineering using our immune-quiet DNA vectors, delivered via a targeted LNP platform. We are establishing a translational path toward off-the-shelf, in vivo-generated cell therapies that eliminate the need for individualized manufacturing. By shifting CAR T therapy from an ex vivo product to an in vivo medicine using our unique DNA-based vectors, this platform has the potential to substantially expand patient access, and we are excited to be advancing it towards first-in-human clinical evaluation." said Hendrik Dietz, CEO of CPTx.
Details of the presentations:
Oral Presentation 341: Programming CAR T cells in vivo with immune-silent ssDNA
Session: In vivo engineering of CAR T cells for autoimmune disease
Presenter: Matthias Bozza
Time: Thursday, May 14, 11:00 AM – 11:15 AM ET
Location: Westin Seaport Grand Ballroom CDE (Concourse Level)
Poster 3084: Targeted lipid nanoparticles deliver immune-silent ssDNA to generate CAR T cells
Time: Thursday, May 14, 05:00 PM – 06:30 PM ET
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)
Poster 3124: ssDNA vectors mitigate DNA-induced inflammation to support non-viral gene delivery
Time: Thursday, May 14, 05:00 PM – 06:30 PM ET
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit Level)
(Press release, CPTx Bio, MAY 4, 2026, View Source [SID1234665070])