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Adeno-associated viral vector serotype 9 containing the human SMN gene

   ZOLGENSMA   
  • INN (International Nonproprietary Name) : onasemnogene abeparvovec
  • Code name : AVXS-101;scAAV9.CB.SMN
  • Chemical name or description : Non-replicating recombinant self-complementary (sc) adeno-associated virus serotype 9 (AAV9) vector containing the cDNA of the survival of motor neuron 2 (SMN2) gene under the control of the hybrid cytomegalovirus (CMV) en hancer/chicken beta-actin promoter (CBA)
  • Type of product : Gene therapy product
  • Type of production : Biotechnology
  • Orphan designation : Europe

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