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Revolutionizing Blood Cancer Therapy: Next-Gen CAR-T Therapy with In Vivo Nanoparticle Targeting

Objective

CAR-T cell therapy has transformed the treatment landscape for blood cancers but remains restricted to a minority of patients due to high costs, lengthy ex vivo manufacturing, and limited specialized hospital capacity. Current therapies cost €300–350k per patient, take weeks to deliver, and suffer from bottlenecks in viral vector manufacturing. Moreover, ex vivo processing drives T-cell exhaustion and raises safety risks, while access remains out of reach for up to 70% of eligible patients.
NanoCell Therapeutics introduces NANOCAR, a first-in-class, non-viral, in vivo CAR-T platform based on proprietary T cell-targeted lipid nanoparticles (tLNPs). This system delivers minicircle DNA encoding a CAR transposon together with short-lived mRNA encoding the Sleeping Beauty transposase, enabling stable genomic integration of CAR constructs directly within circulating T cells. A single administration generates durable CAR-T cells in vivo, bypassing the need for patient cell harvesting, viral vectors, and costly GMP expansion.
The project will advance NCTX-01, our lead clinical candidate, towards first-in-human readiness by: (i) establishing GMP-ready manufacturing of all components, (ii) developing a robust analytical and stability package, (iii) demonstrating safety and efficacy in relevant preclinical models, and (iv) aligning with regulatory authorities. Parallel business objectives include IP strengthening, investor-ready business planning, and preparation for licensing discussions with major pharmaceutical partners.
By achieving TRL 5, NANOCAR will validate a transformative, scalable, and cost-efficient alternative to conventional CAR-T. This innovation has the potential to expand patient access to life-saving therapies, cut treatment costs by up to 99%, and extend beyond oncology into autoimmune diseases and solid tumors. Success will position Europe at the forefront of next-generation cell and gene therapy, driving both health and economic impact.

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Keywords

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Programme(s)

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Topic(s)

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HORIZON-EIC - HORIZON EIC Grants

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Call for proposal

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(opens in new window) HORIZON-EIC-2025-TRANSITIONOPEN

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Coordinator

NANOCELL THERAPEUTICS B.V.
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 2 311 463,75
Address
PADUALAAN 8
3584 CH UTRECHT
Netherlands

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
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Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

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