Alipogene tiparvovec

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Alipogene tiparvovec
Clinical data
Trade names Glybera
AHFS/Drugs.com UK Drug Information
Legal status
  • Prescription only
Routes Intramuscular injection
Identifiers
ATC code C10AX10
UNII 20OK4AFR4Y YesY
Chemical data
Formula ?
Gene therapy using an AAV vector. A new gene is inserted into a cell using the AAV protein shell. The new gene often integrates in a precise location and then makes functional protein to treat a disease.

Alipogene tiparvovec (marketed under the trade name Glybera) is a gene therapy treatment that compensates for lipoprotein lipase deficiency (LPLD), which can cause severe pancreatitis.[1] In July 2012, the European Medicines Agency recommended it for approval, the first recommendation for a gene therapy treatment in either Europe or the United States. The recommendation was endorsed by the European Commission in November 2012[2][3] and commercial rollout is expected in late 2014.[4][5]

Mechanism[edit]

The adeno-associated virus serotype 1 (AAV1) viral vector delivers an intact copy of the human lipoprotein lipase (LPL) gene. Data from the clinical trials indicates that fat concentrations in blood were reduced between 3 and 12 weeks after injection, in nearly all patients. The advantages of AAV include apparent lack of pathogenicity, delivery to non-dividing cells, and much smaller risk of insertion[6] compared to retroviruses, which show random insertion with accompanying risk of cancer. AAV also presents very low immunogenicity, mainly restricted to generating neutralizing antibodies, and little well defined cytotoxic response.[7][8][9] The cloning capacity of the vector is limited to replacement of the virus's 4.8 kilobase genome.

Cost[edit]

Alipogene tiparvovec is expected to cost around $1.6 million for treatment[10] which will make it the most expensive medicine in the world.[11]

See Also[edit]

References[edit]

  1. ^ European Agency Backs Approval of a Gene Therapy July 20, 2012
  2. ^ Gallagher, James. (2012-11-02) BBC News – Gene therapy: Glybera approved by European Commission. Bbc.co.uk. Retrieved on 2012-12-15.
  3. ^ Richards, Sabrina. "Gene Therapy Arrives in Europe". The Scientist. Retrieved 16 November 2012. 
  4. ^ Press Release. UniQure (2012-11-02). Retrieved on 2012-12-15.
  5. ^ "Chiesi and uniQure delay Glybera launch to add data". Biotechnology. The Pharma Letter. August 4, 2014. Retrieved 2014-08-28. 
  6. ^ Valdmanis, PN, Lisowski, L, Kay, MA (November 2012). "rAAV-mediated tumorigenesis: still unresolved after an AAV assault.". Molecular Therapy 20 (11): 2014–17. doi:10.1038/mt.2012.220. PMID 23131853. 
  7. ^ Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J (September 1999). "Immune responses to adenovirus and adeno-associated virus in humans". Gene Therapy 6 (9): 1574–83. doi:10.1038/sj.gt.3300994. PMID 10490767. 
  8. ^ Hernandez YJ, Wang J, Kearns WG, Loiler S, Poirier A, Flotte TR (1 October 1999). "Latent Adeno-Associated Virus Infection Elicits Humoral but Not Cell-Mediated Immune Responses in a Nonhuman Primate Model". Journal of Virology 73 (10): 8549–58. PMC 112875. PMID 10482608. 
  9. ^ Ponnazhagan S, Mukherjee P, Yoder MC, et al. (April 1997). "Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice". Gene 190 (1): 203–10. doi:10.1016/S0378-1119(96)00576-8. PMID 9185868. 
  10. ^ Gene-Therapy Approval Marks Major Milestone
  11. ^ Gene therapy approved in Europe for first time