RARECast: New Means of Regulating Genes May Hold Promise for Rare Disease Patients

April 15, 2016


RaNA Therapeutics is pursuing treatments for rare diseases, such as Spinal Muscular Atrophy and Friedreich’s Ataxia, with a new therapeutic approach that targets a previously unexplored druggable space. We spoke to Ron Renaud, CEO of RaNA, about his company’s effort to selectively upgregulate genes as a way to treat and prevent disease, the challenges in developing such drugs, and why the company has decided to initially target rare diseases with this new technology.

RARECast is a weekly series by Daniel S. Levine. 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, which produces The Bio Report and RARECast podcasts. Read his full bio here. 

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