Rocket Pharmaceuticals focuses on the root cause of rare pediatric diseases

Rocket Pharmaceuticals (Nasdaq:RCKT) is a late-stage clinical biotech focusing on developing gene therapies for rare pediatric diseases with high unmet need.

In 2016, that focus drew in Kinnari Patel, president and COO and Jonathan Schwartz, chief medical officer of the company.

“My passion has been making rare disease drug development more efficient and having more therapies available per patient,” Patel said. “That’s always been my goal since I started my student rotation at the Office of Orphan Product Development at FDA in 2004.”

While safety concerns have recently surfaced in a limited number of gene therapy clinical trials, the field’s promise to develop curative therapies for rare diseases remains captivating.

In fact, Schwartz said the opportunity to work on potentially curative therapies was part of his reason for joining Rocket. “It was a chance to work on cutting-edge science addressing the caus…

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