LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and Wilson disease, conditions characterized by liver damage.
For each condition, when the healthy hepatocytes which carried the corrected gene were expanded, disease markers improved. Parts of or the entire diseased liver were repopulated.
The Data
HT1
HT1 patients don’t have the gene encoding FAH. FAH is necessary to metabolize tyrosine, an amino acid. As a result, toxic metabolites accumulate.
Using HT1 mice models which had liver damage, researchers demonstrated that hepatocytes corrected by GeneRide technology were able to repopulate the full liver. This repopulation occurred just four weeks after administration. All of the diseased hepatocytes were replaced with corrected hepatocytes. Essentially, brand new livers were created in the mice.
The HT1 mice which received the treatment exhibited normal liver function and body growth. Additionally, they had undetectable levels of succinylcholine acetone. Most notably, these mice no longer necessitated the standard of care treatment for HT1.
When directly comparing the standard of care to GeneRide technology, GeneRide had a better reduction in succinylcholine acetone and alfa-fetoprotein. Both of these are biomarkers for the condition.
Wilson Disease
Wilson disease is caused by a buildup of copper in the body. This over-accumulation is toxic and damages tissues.
Using a mouse model, researchers showed that the corrected hepatocytes, created by GeneRide technology, were able to repopulate the liver. The mice had improved liver function, copper levels in their urine, and hepatomegaly.
More information on this data can be read here.
