Gene Therapy Progress for Batten Disease and Sanfilippo Syndrome

REGENXBIO is a biotechnology company that has been developing a technology platform for gene therapy called NAV. AAV9 is one of the vectors being tested with NAV technology as a treatment option for MPS IIIA and B (Sanfilippo syndrome), CLN1 Disease, and Juvenile Batten Disease.

The company has just announced a license agreement with Abeona Therapeutics for the NAV AAV9 vector. Abeona Therapeutics is a biopharmaceutical company that focuses on gene therapy. The agreement between these two organizations validates NAV AAV9 as a future treatment option for lysosomal storage diseases that currently have no approved therapies. With the license, Abeona can now develop and commercialize this therapy worldwide.

NAV AAV9 has proved its potential during clinical and preclinical programs and this licensing agreement should advance clinical trials for this therapy.

The Need for Options

Sanfilippo syndrome and Batten disease patients currently have no options for treatment. In Sanfilippo syndrome, just one, singular missing enzyme causes the disease. Patients experience seizures, loss of vision/speech in addition to other symptoms and their behavior often turns aggressive. Most people with the disease don’t live past the age of 18. Batten Disease patients are missing proteins which causes lipopigment buildup. Patients deal with developmental regression, heart issues, and blindness among other symptoms and similarly to Sanfilippo syndrome, patients pass away far too young.

We need to find these patients more options and we need to find them fast. Gene therapy has shown its potential, and the acceleration of clinical trials is an essential next step.

Looking Forward

The agreement between this biotechnology company, which has found the potential in AAV9, and this pharmaceutical company, which has the power to commercialize treatments, could have a dramatic impact on these patient communities. Keep your eye out for more updates on the AAV9 vector and future clinical trials. Our fingers are all crossed for a speedy development.

You can read more about this agreement, NAV technology, and the AAV9 vector here.

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