The investigative treatment is at preclinical stages of development. The U.S. Food and Drug Administration granted orphan drug status to ABO-201 in 2017.
How ABO-201 works
In juvenile Batten disease, the CLN3 gene is mutated. Sometimes the mutations leads to parts of the gene to be missing. The CLN3 gene provides instructions to make a protein called battenin, which is normally found across cell membranes. Mutations in the CLN3 gene are thought to disrupt the function of battenin. The exact function of battenin protein is not yet understood.
ABO-201 is a type of gene therapy where the intact CLN3 gene is delivered into the cells with the help of a special virus, called adeno-associated virus (AAV). All harmful parts of the virus are removed, and after delivering the CLN3 gene to the cells, the virus dies.
It is hoped that the intact CLN3 gene delivered to the cells in this way will enable them to produce functional battenin protein.
Researchers in the U.S. assessed the effectiveness of ABO-201 treatment in a preclinical study using mice carrying a deletion in the CLN3 gene. This mutation is also common in people with juvenile Batten disease. They treated the mice with a single dose of ABO-201, which significantly restored motor activity to almost the same levels seen in healthy control mice, within one month.
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