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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GSK could pay Arrowhead $1B to develop NASH RNAi therapeutic 

Arrowhead Pharmaceuticals (NSDQ:ARWR) has signed an exclusive license agreement with GlaxoSmithKline (NYSE:GSK) related to Arrowhead’s experimental RNA interference (RNAi) therapeutic ARO-HSD.

The drug is now the subject of a Phase 1/2 study focusing on the drug’s potential as a treatment for nonalcoholic steatohepatitis (NASH), which involves liver inflammation and can cause cirrhosis and liver failure.

ARO-HSD has “the potential to be the first investigational therapeutic to achieve robust reductions in mRNA and protein levels of hepatic HSD17B13, leading to reductions in ALT, a liver enzyme typically elevated in liver diseases such as NASH,” said Christopher Anzalone, Arrowhead’s president and CEO, in a statement.

In exchange for an exclusive license to develop and commercialize the drug throughout the world apart from in China, GSK will make an upfront payment to Arrowhead of $120 million. GSK stands to earn an additional $30 million once the Phase 2…

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