Investor Outlook: Rising from the Ashes; GSK's European Approval of Strimvelis for ADA-SCID

Human Gene Therapy Clinical Development

Volumn 27, Issue 2, 2016, Pages 57-61

  Schimmer, Joshua ^a   Breazzano, Steven ^a  

^a Piper Jaffray Company   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

IMMUNOLOGIC AGENT; RETROVIRUS VECTOR; STRIMVELIS; UNCLASSIFIED DRUG; ADENOSINE DEAMINASE;

ADENOSINE DEAMINASE DEFICIENCY; CLINICAL TRIAL (TOPIC); COMMERCIAL PHENOMENA; DRUG APPROVAL; EUROPE; EX VIVO GENE TRANSFER; HUMAN; NONHUMAN; PRIORITY JOURNAL; REVIEW; VIRAL GENE DELIVERY SYSTEM; AGAMMAGLOBULINEMIA; DEFICIENCY; ECONOMICS; GENE THERAPY; GENE VECTOR; GENETICS; INVESTMENT; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY; THERAPEUTIC USE;

ADENOSINE DEAMINASE; AGAMMAGLOBULINEMIA; DRUG APPROVAL; EUROPE; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INVESTMENTS; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY;

EID: 85006216313     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2016.29010.ind     Document Type: Review

References (10)

1. GSK Pharmaceuticals. GSK press releases. http:// us. gsk. com/en-us/media/press-releases (last accessed May 31, 2016).
2. U. S. National Library of Medicine; Genetics Home Reference. https://ghr. nlm. nih. gov/condition/ adenosine-deaminase-deficiency (last accessed April 27, 2016).
3. Booth C, Gaspar HB. Pegademase bovine (PEGADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Biologics: Targets & Therapy 2009;3: 349-358.
4. Chan B, Wara D, Bastian J, Hershfield MS, Bohnsack J, Azen CG, Parkman R, Weinberg K, Kohn DB. Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clinical Immunology. 2016;177:133-143.
5. European Medicines Agency. Strimvelis opinion. www. ema. europa. eu (last accessed April 27, 2016).
6. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, et al. Gene Therapy for immunodeficiency due to adenosine deaminase deficiency. N Eng J Med 2009;360:447-458.
7. GSK. GSK investor presentation. November 3, 2015.
8. European Medicines Agency. Reflection paper on management of clinical risks deriving from insertional mutagenesis. www. ema. europa. eu (last accessed April 27, 2016).
9. FiercePharma. GSK promises 'bubble boy' gene therapy will not break the bank as new pricing model revealed. www. fiercepharma. com (last accessed April 27, 2016).
10. Financial Times. GSK to allow staggered payments for EMA-approved gene therapy. www. ft. com (last accessed April 27, 2016).