CELTIC-19 granted ATMP classification by EMA

Private UK-based cell and gene therapy company Ixaka, previously known as Rexgenero, has announced 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 hematological 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.