Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

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Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs…

Continue ReadingOverexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

A phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus

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Therapeutic Candidate or Device IMPT-514 is an autologous T-cell product genetically modified to express a chimeric antigen receptor targeting CD19 and CD20 Indication Active, refractory lupus nephritis (LN) and systemic…

Continue ReadingA phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus