LPL Vector Designated an Orphan Medicinal Product

An adeno-associated viral vector (AAV) expressing lipoprotein lipase (LPL) has been designated an orphan medicinal product for the treatment of LPL deficiency by the European Union (EU).

LPL deficiency is a disorder caused by a deficiency of LPL, the principal enzyme involved in the clearance of triglycerides from the plasma. The AAV vector expressing the LPL gene can be injected in the muscles of LPL-deficient patients. After injection, LPL is transported to the cell nucleus. The cell's transcription machinery ‘reads' the LPL gene resulting in the production of the LPL protein. LPL is secreted by the muscle cells and transported into the bloodstream. There, LPL adheres to the wall of a blood vessel where it starts clearing triglycerides.

"LPL deficiency is an ideal target for gene therapy and our preclinical data give us great confidence that we may be able to rapidly provide a treatment in this area of unmet medical need,” stated Dr. Jan Boesen, CEO of Amsterdam Medical Therapeutics (AMT, The Netherlands; www.amtbv.com), which developed the new vector.

Orphan medicinal products are so-named because of the rarity of the diseases they treat. Measures were first put in place to create incentives for developing orphan medicinal products in the United States in 1983. Incentives are required, as there are more than 5,000 identified diseases that affect less than 0.05% of the population and for which there are currently no satisfactory treatments. Japan, Singapore, and Australia have introduced similar programs, as has the EU.





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