Ixaka’s CELTIC-19 granted Advanced Therapy Medicinal Product classification by EMA
Ixaka, an integrated cell and gene therapy company, says that Advanced Therapy Medicinal Product (ATMP) classification has been granted by the European Medicines Agency (EMA) for its Chemically Encapsulated Lentiviral vector for Targeted In Vivo CAR T-cell therapy (CELTIC-19) targeted nanoparticle (TNP) product.
ATMP status was granted to CELTIC-19 due to its potential as a gene therapy medicinal product that targets specific cells and expression of the gene of interest directly within the patient’s body. Such an approach makes it possible to eliminate all the ex vivo stages of genetic modification, that are required for the production of currently marketed cell therapy products.
CELTIC-19 is Ixaka’s lead TNP program for CD19 haematological malignancies. It consists of a polymer nanoparticle encapsulating a bald lentiviral vector encoding for a T-cell specific promoter and the chimeric antigen receptor (CAR). The nanoparticle is coated with a CD3 binding molecule allowing in vivo targeting and transduction of the T-cells. The construct can then be infused systemically into the bloodstream to target and genetically modify T-cells within the body. This approach allows the generation of CAR T-cells which are potentially more efficacious, safer, and considerably less expensive to produce than established CAR T-cell therapies, which have been shown to be effective and have been approved for use in CD19-malignancies.
CELTIC-19’s numerous advantages over established ex vivo CAR T-cells therapies, which include but are not limited to its high specificity and transduction efficiency, retreatment possibility, no cytokine requirement and persistent expression.
Gilbert Wagener, Senior Vice President, Chief Medical Officer at Ixaka commented: “TNP-based in vivo CAR T-cell therapies such as Ixaka’s represent a significant advance over recently approved ex vivo CAR T-cell therapies, and hold the promise of delivering more effective, universal, and safer treatment option for patients. It is great to see this potential recognized by the Europeans Medicine Agency.”
Joe Dupere, CEO at Ixaka commented: “Our nanoparticle-based in vivo gene delivery technology is ideally positioned to deliver on the promise of in vivo CAR-T therapies to transform cancer treatment without the need for costly dedicated manufacturing sites for T-cell modification. The designation of CELTIC-19 as an Advanced Therapy Medicinal Product further signifies its potential as a ground-breaking new treatment option and is an important step on our continued journey.”
