Grant: CIHR Fellowship
University of British Columbia
$135,000.00
Redevelopment and Optimization of an Adeno-Associated Virus Gene Therapy for the Treatment of Lipoprotein Lipase Deficiency
FY 2019-20
Summary
Lipoprotein lipase (LPL) catabolizes the breakdown of triglycerides in blood. LPL is a critical enzyme in the determination of lipoprotein profiles and predisposition to heart disease and stroke. A complete or partial LPL deficiency (type I hyperlipoproteinemia) presents during childhood with high triglycerides, pancreatitis, and a high risk of mortality. We have previously developed a gene therapy treatment for the LPL deficiency and phase I-III clinical trials have demonstrated its long-term safety and effectiveness. In 2012, this drug, Glybera® (alipogene tiparvovec) became the first gene therapy product to receive regulatory approval. However, commercialization was limited due to the extremely high price of the drug (>$1M/patient), in part due to the high cost of smaller-scale production methods. Since 2017, Glybera is no longer marketed. Drugs and enzyme replacement therapies are ineffective and as of now there is no treatment for LPL deficiency. In collaboration with the National Research Council of Canada, this aim of this proposal is to develop, optimize and validate a more cost-effective AAV-based gene therapy variant of Glybera for the treatment of LPL deficiency.
Department
Program
CIHR Fellowship
Recipient
Location
British Columbia, Canada
Recipient Type
Academia
Type
Grant
Source
CIHR Funding Decisions Database
As published
Id
412576
Name
Mehta Neel
Region
British Columbia
Country
Canada
Orgtype
Institution Paid; Research Institution
Instname2
Circulatory and Respiratory Health
Theme2
Biomedical
Prcname2
Fellowships - Post-PhD
Projecttitle
Redevelopment and Optimization of an Adeno-Associated Virus Gene Therapy for the Treatment of Lipoprotein Lipase Deficiency
Programtype2
Fellowship Programs
Orgnameinp2
University of British Columbia
Cihramount2
$135,000
Cihrequipment2
$0
Programname2
CIHR Fellowship
Keyworddelim
Adeno-Associated Viral Vector; Gene Therapy; Genetic Diseases; Hypertriglyceridemia; Lipid Transport; Lipoprotein Lipase; Lipoprotein Lipase Deficiency
Abstract
Lipoprotein lipase (LPL) catabolizes the breakdown of triglycerides in blood. LPL is a critical enzyme in the determination of lipoprotein profiles and predisposition to heart disease and stroke. A complete or partial LPL deficiency (type I hyperlipoproteinemia) presents during childhood with high triglycerides, pancreatitis, and a high risk of mortality. We have previously developed a gene therapy treatment for the LPL deficiency and phase I-III clinical trials have demonstrated its long-term safety and effectiveness. In 2012, this drug, Glybera® (alipogene tiparvovec) became the first gene therapy product to receive regulatory approval. However, commercialization was limited due to the extremely high price of the drug (>$1M/patient), in part due to the high cost of smaller-scale production methods. Since 2017, Glybera is no longer marketed. Drugs and enzyme replacement therapies are ineffective and as of now there is no treatment for LPL deficiency. In collaboration with the National Research Council of Canada, this aim of this proposal is to develop, optimize and validate a more cost-effective AAV-based gene therapy variant of Glybera for the treatment of LPL deficiency.
Competitiondate
201910
Approvedterm2
3 yrs 0 mth
Pinamesdelim
Mehta, Neel
Approvedterm
3 ans 0 mois
Cihrcontribution2
~ $135,000~ $0~
