alipogene tiparvovec + cyclosporine + mycophenolate mofetil

**Alipogene tiparvovec + cyclosporine + mycophenolate mofetil** is a combination regimen used in the context of gene therapy for **familial lipoprotein lipase (LPL) deficiency**. Alipogene tiparvovec is an adeno-associated virus serotype 1 (AAV1)-based gene therapy designed to deliver the human LPL gene variant LPLS447X to muscle tissue, which enables expression of functional LPL enzyme in patients with deficient or non-functional LPL, leading to improved chylomicron clearance and reduction in pancreatitis risk. Cyclosporine (a calcineurin inhibitor immunosuppressant) and mycophenolate mofetil (an inosine monophosphate dehydrogenase inhibitor immunosuppressant) are administered orally to suppress immune responses and prevent immune-mediated loss of transgene expression following alipogene tiparvovec administration. This regimen is used to reduce immunogenicity of the gene therapy vector and ensure maximal transgene expression.

Brand names

Glybera

Other names

alipogene tiparvovec + cyclosporine + mycophenolate mofetil