100 top cell and gene therapy companies to watch in 2023

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The cell and gene therapy sector is poised to deliver a wave of new therapies that could potentially 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, the cell and gene therapy industry continues to offer significant therapeutic potential and commercial opportunities.

This year, we have beefed up our list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.

In selecting the companies for this list, we weighed public companies’ degree of involvement in cell and gene therapy, including drug developers, equipment vendors and CDMOs specializing in the area. For private companies, we factored in their funding levels, pipeline and degree of history for the list.


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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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