Cell and gene therapies are a new category of medicines transforming how we treat and potentially cure disease. Both cell and gene therapies seek to modify genetic material to fight disease. Cell therapies involve cultivating or modifying cells outside the body before injecting them into a patient. Gene therapies use genetic material to manipulate the expression of a gene or alter the properties of an individual’s cells.

Today, seven cell and gene therapies are approved for marketing in the US and fewer than 10 are approved in Europe. Dozens of new cell and gene therapies are expected to become available in the US in the coming years. According to recent research, more than 1,000 cell and gene therapies are currently in development, with 50–75 therapies expected to be approved in the US by 2030.

In response to this growing pipeline and the hope these therapies offer, FasterCures convened roundtables and discussions to bring to light the unique regulatory and access considerations posed by cell and gene therapies. On November 3, 2021, FasterCures brought together its stakeholder community, along with Peter Marks, director of CBER at FDA, in a virtual roundtable. The goals of the roundtable included taking stock of the current status of cell and gene therapies given the disruptions caused by the COVID-19 pandemic, and identifying opportunities for collective engagement in 2022.

This report summarizes the key takeaways from the November 3 roundtable, and considers the developments that will take place in cell and gene therapies in 2022.