LEXEO programs in development
Batten Disease (CLN2)
Autosomal recessive lysosomal storage disease with less than 1000 cases worldwide. Caused by mutation in the CLN2 gene and results in cognitive impairement, visual failure, seizures, and deteriorating motor development. LXc-004 is an AAV mediated gene therapy treatment delivering CLN2 to the central nervous system.
APOE4 Alzheimer’s Disease
Alzheimer’s disease is the leading cause of late life mental failure in humans. APOE is the major transporter of cholesterol in the brain and its role in the pathogenesis of Alzheimer’s disease is linked, in part, to development of amyloid plaques and tau-tangles. Inheritance risk for APOE4 results in more frequent and earlier age of onset for developing Alzheimer’s disease while inheritance of APOE2 has the reverse effect. LXc-001 is an AAV mediated gene therapy treatment delivering APOE2 to the central nervous system.
Other LEXEO Programs
for the treatment of Metachromatic leukodystrophy
LXc-002 and LXc-003,
"Vectorized” antibody programs addressing Tauopathies including Alzheimer’s disease and Chronic traumatic encephalopathy.
LEXEO holds an exclusive option to research, develop and commercialize three late pre-clinical stage gene therapy product candidates in the liver-directed and cardiac hereditary disease areas, as well as an option on eight pre-clinical “vectorized” antibodies in the inflammation/allergy and oncology therapeutic areas addressing acquired diseases.