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Biotech company battles medical mysteries in Cleveland

Posted at 4:09 PM, Nov 09, 2017

A Cleveland-based biotechnology company is expanding and hoping to make their life-changing gene therapy solutions more widely available.

Abeona Therapeutics can be found in a converted warehouse on E. 65th and Carnegie in midtown Cleveland. The building was once a car painting facility, then a printing shop - now it's filled with labs.

Inside those labs, science fiction is slowly becoming reality. There is a commitment to patients — and a commitment to Cleveland.

“Cleveland’s been a great biotech hub,” said CEO Timothy Miller. “It’s biotech on the North Coast.” 

Research and development, which are more typical for cities like San Francisco and Boston, have a home in Cleveland’s health-tech corridor. In that corridor, Abeona is focused on finding gene therapy solutions for six of 7,000 rare diseases.

“Next-generation rare disease gene therapies and cell therapies for kids with rare genetic conditions,” Miller said. “It’s very Star Trek in some respects.”

Michelle Berg is the vice-president of patient advocacy. She said by and large, they are focused on diseases that impact children and are ultimately fatal. 

“What we’ve heard from the families is that we brought them hope — this possibility where nothing existed before. These are rare diseases where there is nothing even in preclinical development,” Berg said.

These diseases include Sanfillipo syndrome, batten disease, fanconi anemia, and epidermolysis bullosa.

Within the next six months, Abeona plans to complete an expansion, so in addition to labs, there will also be commercial manufacturing — the first of its kind for biotech in Ohio. The company plans to hire an additional 25 to 40 employees to go along with this expansion.