Cleveland Company Developing Rare Therapies for Children


Abeona Therapeutics further solidifying Cleveland’s stronghold in the biotechnology and gene therapy fields.

The pharmaceutical industry, responsible for the development, production and marketing of medications, is a $450 billion industry in the United States. This critical, complex machine leads to breakthrough discoveries of cures and overall advances in global health care, but the space is dominated by only a few major corporations focused on a few major diseases. Abeona Therapeutics Inc. in Cleveland is making a name for itself by focusing narrowly on life-threatening rare diseases affecting children—and accelerating the path towards cures.

“Large pharmaceuticals focus on the most common diseases. In that model, you’re focused on serving the broadest patient populations and maximizing profitability, so it can be a challenge to concentrate resources on rare diseases,” said Steve Goden, Director of Finance and Operations at Abeona. “Our founders are very passionate about kids and very knowledgeable — pioneering even — in gene therapy. We saw the potential of the scientific discovery and its commercial translation as the perfect combination for our team to make a difference.”

A disease is characterized as rare if it affects fewer than 200,000 people in the United States. While rare diseases can affect any age group, about 50% of people impacted are children—accounting for 35% of deaths in the first year of life. Over 95% of rare diseases do not have a single FDA or EMA approved drug treatment. Abeona, named after the Roman Goddess who protects children as they take their first steps away from home, set out to address this disparity.

The publicly-traded company (NASDAQ: ABEO), founded in Cleveland in 2013, has grown quickly thanks to early breakthroughs around three rare diseases, including Sanfilippo Syndrome, Recessive Dystrophic Epidermolysis Bullosa and Infantile Batten Disease. Abeona has offices in Dallas, New York City and Cleveland. In late 2017, the company announced the development of a commercial gene therapy manufacturing facility, the Elisa Linton Center for Rare Disease Therapies, named after a patient whose parents drove interest and fundraising for a cure.

By developing their own production center in Cleveland’s emerging Health-Tech Corridor, Abeona will be able to better control their production timeline. Currently, there are very few companies that can manufacture the materials Abeona needs for clinical trials, so the pace of their therapy development was often dependent on others. With a state-of-the-art facility in Cleveland, they can now control their own production timelines.

Abeona’s success is part of a larger movement that has seen Cleveland cement itself as a leading center for biotechnology innovation and investment in recent years, attracting more than $2.17 billion in biotechnology funding in 2016. In addition to accessible capital, northeast Ohio is also home to leading healthcare institutions and research centers including the Cleveland Clinic, University Hospitals, the HealthTech Corridor and BioEnterprise, a northeast Ohio regional partner of Ohio Third Frontier.

“We think this community, with premier academic, biotechnology and healthcare institutions, is positioned to become an anchor in the gene therapy space. We’re hopeful that as others realize what we’re able to do here in Cleveland, they’ll consider establishing operations in the same neighborhood,” said Goden. “We can all work together and can benefit from one another. We can all win.”

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