NanoCAR: Advancing In Vivo CAR T Therapy

A Scalable Approach to Reprogramming T Cells Inside the Body

The NanoCAR project is developing an in vivo CAR-T therapy approach based on targeted lipid nanoparticles, or tLNPs. The technology is designed to deliver genetic material directly into T cells within the patient, removing the need to extract, genetically modify, and reinfuse the patient’s cells.
By moving T-cell reprogramming from an external manufacturing facility into the body “in vivo”, NanoCAR aims to make CAR-T therapy more accessible, scalable, cost-efficient, and safer.

Project Rationale

Addressing the Limitations of Current CAR T Therapy

CAR-T therapies have demonstrated significant clinical success in the treatment of blood cancers. However, their reach remains limited by complex manufacturing, high costs, lengthy treatment timelines, and the need for specialized treatment centers.

Current challenges include:

Treatment costs €300,000 to €1 million per patient
Limited access due to specialized hospital requirements and capacity constraints
Treatment delays caused by complex manufacturing timelines
Safety concerns, including cytokine release syndrom

As a result, many eligible patients are unable to receive treatment. NanoCAR is being developed to address the need for a more accessible, affordable, scalable, and safer approach.

NanoCAR Technology

Delivering CAR T-Cell Reprogramming In Vivo

Unlike conventional CAR-T therapies, which require ex vivo extraction and modification of a patient’s T cells, the NanoCAR approach is designed to genetically reprogram T cells directly in vivo.

The technology combines four principal components:

Targeted lipid nanoparticles for the delivery of nucleic acids
Minicircle DNA encoding CAR constructs
Messenger RNA encoding a transposase for stable genome integration
Targeting and activating ligands designed to support specificity for T cells

This non-viral delivery approach is intended to eliminate the need for complex ex vivo manufacturing while supporting the development of a scalable and cost-efficient gene therapy.

Project Objectives

Advancing NanoCAR Toward Clinical Translation

The NanoCAR project is designed to move the technology from early proof-of-principle results toward a lead clinical candidate and validation at Technology Readiness Level 5, or TRL 5.

The work is organized around three connected objectives:

Objectives

Project Impact

Expanding Access to More Scalable and Patient-Friendly CAR T Therapy

NanoCAR is designed to address both the clinical limitations of current CAR T therapy and the broader challenges associated with cost, access, and healthcare delivery.

The expected impact can be viewed across two connected areas:

Impact

Advancing a More Accessible Approach to CAR T Therapy

NanoCAR represents a paradigm shift from complex, expensive ex vivo CAR T therapies toward a simple, scalable, in vivo gene therapy approach.

By addressing major clinical and logistical barriers, the project aims to deliver a next-generation, accessible cancer treatment with far-reaching clinical and societal benefits.

Contact Details

Head Office:

NanoCell Therapeutics Inc.
1055 Westlakes Drive, 3rd floor
Berwyn, PA 19312
United States

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