Gene therapy from Abeona Therapeutics passes early test in first clinical trial

Story excerpt provided by Crain’s Cleveland Business.

Written by Chuck Soder.

A gene therapy drug developed by Abeona Therapeutics has passed one early test.

The first two patients to receive the therapy appear to be tolerating it well 30 days after being treated — and some early evidence suggests it might actually work, according to a news release from Abeona, which has offices in Cleveland and New York.

The drug is designed to treat patients with a fatal degenerative disease called MPS IIIA. It’s also known as Sanfilippo Syndrome Type A.

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Originally published August 2, 2016.

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