alipogene tiparvovec

Alipogene tiparvovec is a gene therapy designed to treat familial lipoprotein lipase deficiency (LPLD), a rare inherited disorder caused by mutations in the LPL gene, which can lead to severe or recurrent pancreatitis. The therapy uses an adeno-associated virus serotype 1 (AAV1) vector to deliver the human LPLS447X gene variant into muscle tissue via intramuscular injection. This enables expression of functional lipoprotein lipase, aiming to restore normal fat metabolism and reduce triglyceride levels in patients with LPLD. Alipogene tiparvovec was developed as the first approved gene therapy for this indication in Europe but was later withdrawn from the market due to commercial challenges and limited patient population[1][2][3][5][6].

Brand names

Glybera

Other names

alipogene tiparvovec