Local company trying to save lives of those fighting rare disease

Monica Robins, WKYC 7:08 PM. EST March 03, 2017

(Photo: Monica Robins, WKYC)

CLEVELAND - Earlier this week, AbeonaTherapeutics, a local biotech company, rang the closing bell on the stock market.

Tuesday was Rare Disease Awareness Day and the company was chosen because of its work. They are giving hope to families fighting diseases that have no treatments or cures.

Inside Abeona Therapeutics, scientists don't have to look far for inspiration. They just have to look on the wall, especially in a little corner of the office meant for visiting kids with a deadly rare disease called Sanfilippo.

"They are usually diagnosed between the ages of two and six and that leads to profound neurological and neuromuscular deficits," according to Abeona Theraputics President and CEO Dr. Timothy Miller. "70 percent don't live to 18."

They're working on gene therapy, using a virus to deliver the correct DNA into each cell with a simple injection.

"We are the only trial in the world right now enrolling patients in this type of disease category," says Dr. Miller.

There are 7,000 rare diseases of which 95% have no treatment or cure. While Sanfilippo is their main focus, the science could one day be a breakthrough.

"Gene therapy is all about delivery and trying to demonstrate that these are actual ways to treat some of these diseases, " explains Dr. Miller. "These will be applicable to many other diseases."

And business is booming. Abeonais looking for more employees to fill these desks. And they just look out their window for possible expansion sites to house a 20,000 square foot manufacturing facility.

Says Dr. Miller, "We're helping people see the possible by bringing jobs to Northeast Ohio, and bringing science into clinical projects. We're hoping to be one of the first gene therapy products in the world."

When Abeona went public in 2015, the company grew 500%. This year, they're looking to expand and have openings for highly skilled people with advanced degrees in biology or chemistry for medical manufacturing positions, clinic trial operations, and program management.

They often recruit from across the country so they're helping Northeast Ohio experience the brain gain.

( 2017 WKYC)

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