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

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.