ABO-201, also known as scAAV-CLN3, is an AAV-based gene therapy being injected into the blood stream once. It is being developed by the pharmaceutical company Abeona Therapeutics for the treatment of juvenile Batten disease (JBD) or CLN3 disease, a fatal lysosomal storage disease of the nervous system caused by autosomal recessive mutations in the CLN3 gene also known as battenin.

How ABO-201 works

The drug uses a virus called adeno-associated virus or AAV to deliver a healthy copy of the CLN3 gene to the body, allowing the production of healthy CLN3 protein. The treatment is not able to reverse all of the effects of the CLN3 mutation.

JBD is characterized by stunted growth, impaired intellect, loss of vision that can lead to blindness, and seizures. Part of the CLN3 gene is deleted in 90 percent of JBD patients, which leads to the production of an abnormally small and dysfunctional CLN3 protein.

The genetic mutation also leads to an accumulation of cell structures called lysosomes, whose function is to destroy cell residue. For a reason that remains unknown, nerve cells are sensitive to the accumulation of lysosomes — and this leads to the symptoms of JBD.

ABO-201 studies

In preclinical studies conducted in mouse models of JBD, ABO-201 was found to reduce nerve inflammation, improve movement, and reduce lysosome accumulation. The results were published in the September 2016 issue of the Journal of Neuroscience.

In January 2017, the European Medicine Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted orphan drug designation to ABO-201 for the treatment of JBD, providing Abeona with incentives and benefits in the European Union, including reduced fees and protection from market competition once ABO-201 is approved.

Note: Batten Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.