Gene therapy has the promise of radically changing the landscape for rare disease patients who are 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.

Daniel S. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013 to provide strategic communications to life sciences companies. He is host of The Bio Report and RARECast podcasts, a senior fellow at the Center for Medicine in the Public Interest, and a member of the advisory board of the California Biotechnology Council.

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