The cell and gene therapy sector is poised to deliver a wave of new therapies with the potential to cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023. While manufacturing and regulatory challenges remain,…
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…