Abeona Therapeutics, a small biotech startup company that develops therapies for lysomal storage diseases, earned the Global Gene’s Champion of Hope award, along with its partners, for its work in developing therapies for children with Sanfilippo Syndrome, a rare genetic disorder.

Abeona, founded earlier this year in Cleveland as a spinoff of Columbus-based Nationwide Children’s Hospital, is developing two products that came out of research at Nationwide. ABX-A and ABX-B have the potential to cure Sanfilippo Syndrome.

Children with Sanfilippo are missing an essential enzyme for normal cellular function, causing toxins in their brains and bodies to cause severe disabilities. Symptoms often appear in the first year of life, causing progressive muscular and cognitive decline, and the disease is usually fatal by the early teens.

Current studies show that a single dose of Abeona’s treatments prompted cells to produce the missing enzymes and help repair the damage.

“What’s unique about this drug is that it’s a collaboration between Abeona, the hospital and eight international foundations to find a treatment for Sanfilippo Syndrome,” says Abeona president and CEO Tim Miller.

Abeona’s products are a result of 10 years of research done by Nationwide’s Haiyan Fu. Abeona is currently raising money to conduct phase I and II clinical trials in 2014. “One of the things that drew me to this company is the pre-clinical data for these drugs,” says Miller. “The life span is improved. If this transfers over to the kids I’ll be very excited.”

While there are no approved treatments for Sanfilippo Syndrome yet, Miller says a number of companies are working on therapies.

Source: Tim Miller
Writer: Karin Connelly