Abeona Advancing Pipeline of Gene Therapies for Rare Diseases

Gene therapy has the promise of radically changing the landscape for rare disease patients without therapeutic options today. Abeona Therapeutics, which is building a pipeline of gene therapies, is not timid in its targets, which includes the progressive neurodegenerative disorders Sanfilippo syndrome types A and B as well as Batten disease. We spoke to Tim Miller, CEO of Abeona, about gene therapy, the company’s pipeline, and the challenges of delivering these therapies to where they are needed within the body.