Investor Outlook: Rising from the Ashes; GSK's European Approval of Strimvelis for ADA-SCID.
GlaxoSmithKline's (GSK) and partner San Raffaele Telethon Institute for Gene Therapy's recent positive European approval for Strimvelis for treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) represents the second EU-approved gene therapy and the first γ-retrovirus and first ex vivo gene therapy. In this article we discuss the significance and implications of this historic approval for the broader gene therapy field.
Piper Jaffray & Co., New York, New York.
Joshua
J
Schimmer
Piper Jaffray & Co., New York, New York.
Steven
S
Breazzano
D000243:Adenosine Deaminase / Q000172:deficiency* / Q000235:genetics; D000361:Agammaglobulinemia / Q000235:genetics / Q000628:therapy*; D017277:Drug Approval; D005060:Europe; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D007449:Investments / Q000191:economics*; D012190:Retroviridae / Q000235:genetics*; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*
D000243:Adenosine Deaminase