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Atidarsagene autotemcel

Description

Atidarsagene autotemcel is an ex vivo genetically modified autologous CD34+ hematopoietic stem and progenitor cell (HSPC) gene therapy. Autologous CD34+ HSPCs are collected from patient bone marrow (BM) harvest or from mobilised peripheral blood (mPB) and transduced with a lentiviral vector (ARSA LVV), which inserts one or more copies of the human ARSA complementary deoxyribonucleic acid (cDNA) into the cell’s genome, so that genetically modified cells become capable of expressing the functional ARSA enzyme.

Medicine classification

ATC Code
Title
Class
A16AB21
Atidarsagene autotemcel
A Alimentary tract and metabolism → A16 Other alimentary tract and metabolism products → A16A Other alimentary tract and metabolism products → A16AB Enzymes

Medicines

Drug
Countries

Lithuania

Product monographs

Monograph
Type
Country
LIBMELDY Dispersion for infusion
MPI, EU: SmPC