Gene Therapy: Delivering on the Promise

Story excerpt provided by PharmaVOICE.

Contributed by Denise Myshko.

It’s been 16 years since tragedy struck in the clinical research world. A patient in an experimental gene therapy trial died just days after receiving the therapy. In 1999, Jesse Gelsinger came to the University of Pennsylvania with a rare metabolic disorder that prevents the body from breaking down ammonia. In this trial, a weakened adeno virus, a cold virus, was used as a vector to deliver a corrective gene. But after he received the therapy, Jesse’s body began to shut down and he died a few days later.

Jesse’s death sparked new regulatory controls in gene therapy trials. Early in 2000, the Food and Drug Administration and the National Institutes of Health enhanced patient protection through two new programs: the Gene Therapy Clinical Trial Monitoring Plan and the Gene Transfer Safety Symposia…

…“There is now a broad level of support for adeno-associated virus therapies that can deliver the correct functioning gene to multiple target tissue,” says Tim Miller, Ph.D., CEO, Abeona Therapeutics. “The key with gene therapy is matching the right delivery vehicle for the targeted tissue. 2016 is set up to be a very exciting year with multiple gene therapy companies reporting clinical data. Next year, we’ll be talking about significant insights into the promise of multiple approaches to gene therapies in targeting tissues and correcting underlying disease, many of which have no standard of care treatment.”

Abeona’s lead program is an adeno-associated virus-based gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB), a metabolism disorder. Trials for Sanfilippo types A and B are expected to begin toward the end of 2015 in the United States and in Europe in 2016…

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Originally published September 2015.