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Lysosomal Storage Disorders: Gene Therapy And FDA Orphan Designations

August 29, 2014

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No gene therapy has ever been approved for sale in the United States. In Europe, the EMA (European Medicines Agency), has given regulatory approval to UniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD).

This is the twelfth Blog Post in a continuing series that examines Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post identifies gene therapies for LSDs that have received FDA Orphan Drug Designation in 2013 – 2014.

FDA Orphan Designated Gene Therapies For LSDs (2013 – 2014) 

Drug Name Sponsor Company Indication FDA ODD Date
AAV-G6Pase vector GlyGenix Therapeutics Glycogen storage disease type Ia 03.11.13
Recombinant adenovirus vector AAV2/rh8 expressing human B-hexosaminidase A & B subunits National Tay-Sachs & Allied Diseases Association (NTSAD) Tay-Sachs Disease 03.25.13
Recombinant adeno- associated virus vector AAV2/rh8 expressing human B-hexosaminidase A and B subunits National Tay-Sachs & Allied Diseases Association (NTASD) Sandhoff Disease 03.25.13
Adeno associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs Lysogene (France) MPS III A (Sanfilippo Syndrome) 05.06.13
Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase AbeonaTherapeutics MPS III A (Sanfilippo Syndrome) 04.29.14
Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase Abeona Therapeutics MPS III B (Sanfilippo Syndrome) 04.30.14

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Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons..

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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