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Strimvelis (2696273, GSK2696273)

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Company GlaxoSmithKline plc
DescriptionRetroviral vector delivering adenosine deaminase (ADA) for ex vivo hematopoietic stem cell therapy
Molecular Target Adenosine deaminase (ADA)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector
Latest Stage of DevelopmentApproved
Standard Indication Metabolic (unspecified)
Indication DetailsTreat severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency (ADA-SCID)
Regulatory Designation
PartnerFondazione Telethon;
San Raffaele Hospital

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