Date: 2015-04-06
Type of information: Initiation of development program
phase:
Announcement: initiation of the development program
Company: uniQure (The Netherlands) Chiesi Farmaceutici (Italy)
Product: Glybera® (alipogene tiparvovec)
Action
mechanism: gene therapy. Glybera® is an adeno-associated viral vector (AAV1) based gene therapy, administered intramuscularly (IM) at multiple-sites in a single session. AAV1 carrying the human variant LPLS447X gene is delivered to skeletal muscle, where it becomes active. The LPL protein is expressed and transported to the capillary endothelium where it binds to chylomicrons and VLDL. Alipogene tiparvovec is intended as a curative measure for patients with LPLD and, as well as enhancing chylomicron metabolism, may prevent episodes of pancreatitis.
Disease: lipoprotein lipase deficiency (LPLD)
Therapeutic area: Genetic diseases - Rare diseases
Country:
Trial
details: * On April 6, 2015, uniQure provided an update on multiple gene therapy programs. In early 2016, the Company expects to commence an additional clinical evaluation of Glybera® (alipogen tiparvovec) to be included in a future BLA submission with the FDA. The clinical trial will include next-generation manufacturing process enhancements, which are currently being implemented.
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