Gene and cell therapy companies have raised $2.3 billion in equity in 2018 as of the end of last week, according to BioCentury’s BCIQ database. The figure is more than half of the $4.5 billion raised in all of 2017.
Gene and cell therapies companies’ annual fund-raising totals have increased each year since 2008. The biggest jump was in 2015, when equity raised jumped to $3.3 billion from $1.6 billion in 2014.
FDA Commissioner Scott Gottlieb tweeted on Tuesday that there are more than 500 active INDs involving gene therapy products in the U.S., over 100 of which the agency received in 2017. “This shows the intensity of scientific work going on in this field,” he said.
He also tweeted that FDA has received 62 submissions for regenerative medicine advanced therapy (RMAT) designation, of which FDA has granted 19. Among those that have received the designation, 14 therapies also have Orphan Drug designation.
The RMAT designation, which was introduced in 2016 as part of the 21st Century Cures Act, applies the principles pioneered in FDA’s breakthrough designation to regenerative therapies, including cell therapies and some gene therapies. RMAT commits FDA to collaborate with sponsors early in the product development cycle to design clinical trials, address quality and manufacturing issues and delineate a streamlined path to an approval decision (see BioCentury, Dec. 15, 2017).
Gottlieb announced Tuesday that FDA will soon release a framework to facilitate manufacturing of gene therapies, and will lay out development strategies for gene therapies to treat specific diseases (see BioCentury Extra, May 22).