Therapeutic Asset Ratings

Autologous Bcma/Gprc5D Dual-Target Car-T

Autologous Cd19/Cd20 Dual-Target Car-T Module

Autologous Cd19/Cd20 Dual-Target Car-T Module

Autologous Cd19/Cd22 Dual-Target Car-T Module

Autologous Cd19/Cd22 Dual-Target Car-T Module

Autologous Cd33/Cd123 Dual-Target Car-T Module

Autologous Cd33/Cd123 Dual-Target Car-T Module

Autologous Cd33/Cll1 Dual-Target Car-T Module

Autologous Cd33/Cll1 Dual-Target Car-T Module

Autologous Cd5/Cd7 Dual-Target Car-T Module

Autologous Cd5/Cd7 Dual-Target Car-T Module

Autologous Cd7 Car T-Cell

Autologous Cd7 Car T-Cell

Autologous Hucd19 Car T Cells

Autologous human CD34+ hematopoietic stem/precursor cells (HSPCs), obtained from peripheral blood by leukapheresis from plerixaformobilized sickle cell disease (SCD) patients or granulocyte colony stimulating factor (G-CSF) and plerixafor-mobilized transfusion dependent betathalassemia (TDT) patients, genetically modified ex vivo by CRISPR/Cas12a (clustered regularly interspaced palindromic repeats/modified Acidaminococcus sp. Cas12a) endonuclease complexed with a guide RNA (gRNA) that targets the CCAAT-box region of both gamma globin gene (HBG1 and HGB2) promoters on chromosome 11, creating indels that disrupt repressor binding and increase gamma globin expression. The editing components are introduced into the target cell population as a ribonucleoprotein complex by electroporation. The cell suspension is enriched for CD34+ cells using magnetic bead separation. Following electroporation, the cells are cultured in media containing thrombopoietin, Fms-related tyrosine kinase 3 ligand (Flt3L), and stem cell factor (SCF). The substance consists of cells with ?70% CD34/CD45+ purity and ?70% on-target editing.

Autologous T cells obtained from peripheral blood mononuclear cells by leukapheresis, transduced with a self-inactivating, nonreplicating lentiviral vector, encoding a T cell chimeric antigen receptor targeting CD19. The expressed transgene comprises a CD8? leader sequence, a murine anti-CD19 single chain variable fragment (scFv) derived from the mouse hybridoma FMC63, a CD8? hinge and transmembrane region, and a 4-1BB (CD137) and CD3? (TCR?) signaling domain and is under control of the elongation factor 1 alpha (EF1?) promoter. The construct is flanked by 5' and 3' long terminal repeats (LTRs) and also contains a ? packaging signal, a Rev response element (RRE), a central polypurine tract (cPPT) sequence and an optimized Woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The leukapheresis material is enriched for CD4/CD8 T cells by positive immunoselection, activated by CD3 and CD28 agonists and transduced with the vector. Without further cell propagation, the T cells are washed, formulated for infusion, and cryopreserved. The substance is composed of ?80% T cells and ?1% B cells, with a mixture of transgene positive (>3.4%) and negative T cells. The CD4+ and CD8+ naïve T cell subsets (CD45RA+CCR7+) present in the leukapheresis material are largely retained

car19tif cells

Car-20/19-T Cells

Car.70/Il15-Transduced Cb-Nk Cells

cd123-car-nk cells

CD123-CAR T

cd123-cd33 ccar t cells

cd19car-cd3zeta-4-1bb-expressing autologous t-lymphocyte cells

cd19- specific car engineered autologous t-cells

cd19 specific chimeric antigen receptor t cell