Study shows potential of NanoCell Therapeutics’ NCtx platform, and durable CAR-T therapy without ex vivo manufacturing
First indication of stable, functional CAR-T cell generation in vivo using targeted lipid nanoparticles for DNA delivery
WAYNE, Pa., & UTRECHT, Netherlands--(BUSINESS WIRE)--NanoCell Therapeutics, Inc. (“NanoCell”), a biotechnology company developing a non-viral, DNA-based in vivo gene therapy platform, today announced the publication of research data in the Journal for ImmunoTherapy of Cancer demonstrating its novel targeted lipid nanoparticle delivery system, termed NCtx. This technology enables the direct in vivo generation of chimeric antigen receptor T-cells (CAR-T), potentially transforming the accessibility and scalability of CAR-T therapies for cancer patients worldwide.
The study, titled “T cell-specific non-viral DNA delivery and in vivo CAR-T generation using targeted lipid nanoparticles,” represents the first successful demonstration of efficient non-viral DNA delivery to T cells in vivo, positioning NCtx as a promising advancement in the field of in vivo CAR-T therapy.
While CAR-T therapy has shown remarkable success in treating hematologic malignancies, current approaches face significant barriers including high manufacturing costs (approximately $300,000 per patient), complex logistics, lengthy manufacturing times, and limited patient access due to specialized facility requirements. The NCtx platform co-delivers minicircle DNA encoding a CAR construct and transposase mRNA in a single targeted nanoparticle, enabling stable genomic integration and durable CAR expression. The system utilizes dual CD7/CD3 targeting to achieve T cell specificity while activating cells for enhanced DNA uptake.
Researchers demonstrated that a single intravenous dose of NCtx led to robust CAR-T cell generation in multiple humanized mouse models, effective tumor control and significantly extended survival, durable CAR expression and functional anti-tumor activity at doses as low as 4 µg/kg.
“This research demonstrates a new path forward for CAR-T therapy,” said Dr. Jacek Lubelski, senior author and CTO at NanoCell Therapeutics. “By enabling in vivo CAR-T generation through a single, targeted nanoparticle injection, we can potentially dramatically simplify treatment protocols while expanding access in the future to patients worldwide who currently cannot benefit from conventional CAR-T therapies.”
Results were generated using a dual-CAR construct targeting CD19 and CD22, developed by Shanghai Cell Therapy Group (SHCell) https://www.shcell.com/.
"Our collaboration with SHCell underscores the power of combining innovative delivery platforms with cutting-edge CAR designs,” said Dr. Maurits Geerlings, CEO and President of NanoCell Therapeutics. “We are excited about the potential of this partnership to accelerate the development of transformative therapies for patients with B cell cancers and beyond.”
The platform’s modular design enables rapid adaptation for multiple therapeutic targets across oncology and autoimmune diseases. The study was conducted by research teams at NanoCell Therapeutics and the University Medical Centre Utrecht, with support from the European Union’s Horizon Europe and Horizon 2020 research programs.
As NanoCell continues to advance its NCtx platform, the company is expected to pursue an IMPD application pending financing, marking a critical step toward clinical development.
About NanoCell Therapeutics, Inc.
NanoCell Therapeutics is a privately held, transatlantic biotechnology company with locations in Wayne, Pennsylvania, and Utrecht, the Netherlands. The company is dedicated to pioneering transformative in-vivo cell engineering through its non-viral, DNA-based gene therapy platform, primarily targeting oncology and autoimmune diseases.
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