Table 1:

Indications for LCAT activators

Disorder	Mutation Type Or Defect	rhLCAT, WT	rhLCAT, modified	Activating Antibody	Gene Therapy	Peptides	Small Molecules
FLD	Active Site	+	+	−	+	−	−
FLD	Major Truncation	+	+	−	+	−	−
FLD	Small Truncation	+	+	−/+	+	−/+	−/+
FLD	Secretion defects	+	+	−	+	−	−
FLD	Stability/ Structure	+	+	−/+	+	−/+	−/+
FLD	Partial loss of activity	+	+	−/+	+	−/+	−/+
Cholestasis	All types	+	+	+	+	+	+
Atherosclerosis	Low HDL	+	+	+	+	+	+
ACS	Endothelial NO production	+	+	+	+	+	+
Oxidative stress disorders	Oxidative inactivation of endogenous LCAT	+	+	+	+	+	+

LCAT enzyme replacement therapy and gene therapy would not require the presence of functional LCAT. Activating antibodies, peptides and small molecule activators of LCAT require expression of LCAT protein, either wild-type or with a mutation permissive for enzyme activation by the activating agent.