Unlocking Potential Cures For Rare Diseases With Gene Therapy - Dr. Gaurav Shah, CEO of Rocket Pharma

Eighty percent of rare diseases are caused by genetic mutations, which is why many of our recent guests have highlighted the importance of pursuing gene therapies as potential treatments and cures. That’s why we’re particularly pleased to have Dr. Gaurav Shah on Raise the Line today. He’s the CEO of Rocket Pharma, a company that’s in hot pursuit of developing curative gene therapies for patients with inherited genetic diseases, and showing remarkable results in some cases. For instance, a gene therapy for a rare and fatal heart condition called Danon disease is moving every parameter in the right direction, a result Dr. Shah is understandably proud of. “When gene therapy works, it really works. We were able to demonstrate the power of gene therapy for heart disease for the first time in our species,” he tells host Michael Carrese. Don’t miss this fascinating look at the art, science and practicalities of developing gene therapies and where Rocket Pharma is seeing progress. You’ll also learn about the many career options in drug development and hear how Dr. Shah’s background as a Grammy-winning artist informs his approach to leadership.

Mentioned in this episode: https://rocketpharma.com/

If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast