Abeona Therapeutics opens gene therapy manufacturing, immediately starts on expansion

Abeona Therapeutics has opened a 6,000-square-foot gene manufacturing facility in Cleveland, Ohio. (Abeona Therapeutics)

Abeona Therapeutics, another biotech working to develop one-shot gene and cell therapies for rare diseases, is ramping up its manufacturing in anticipation of its getting some of them to market.  

The New York-based developer has opened up what it calls the Elisa Linton Center for Rare Disease Therapies in Cleveland, Ohio, which includes a 6,000-square-foot manufacturing facility. It will provide clinical stage material but also help with commercial production, including for its recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome work. It employs about 20 people.

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The company also has started work on an second phase, 20,000 square foot facility that will be used to boost its commercial production. That will add another 50 workers when complete, an Abeona spokesperson said in an email.

The cost for the facility was kept under wraps, but the company said the new facility should run about one-fourth the cost of a biotech manufacturing facility built in the biotech hubs of Boston or San Francisco.

Abeona, with a market cap of $670 million, is working on a series of trials, including a gene therapy for the lysosomal storage disease Sanfilippo B, ABO-101 and ABO-102 for related lysosomal storage disease Sanfilippo A/MPS IIIA, which has shown some promising initial data in early clinical tests.

RELATED: Abeona says it will start Sanfilippo B gene therapy trial in Q2

Its one-shot gene therapy approach to Sanfilippo A/B and other lysosomal storage diseases diverges from the approach taken by other companies, which have often focused on developing enzyme replacement treatments that would have to be dosed on an ongoing basis.

Also with an eye on its Abeona its commercial future, Abeona last month brought on industry veteran Carsten Thiel as its CEO. His experience includes work at Roche, Amgen and, most recently, Alexion, where he was EVP and chief commercial officer.