familial hypercholesterolemia program

VRX-LDLR

LeXis gene therapy (familial hypercholesterolemia)

Familial amyloidotic polyneuropathy RNAi therapy(BIOALVO)

EBV-TCR-T cells (allogenic, EBV-associated hemophagocytic lymphohistiocytosis/EBV infection)

Adeno-Associated Virus serotype 2 therapy (familial dysautonomia)

ABCB4 gene therapy (lipid nanoparticles, progressive familial intrahepatic cholestasis type 3)

Emapalumab(Gamifant)

Rx

Volanesorsen(Waylivra)

Mipomersen(Kynamro)