Vertex is a leader in the market of CFTR modulators for cystic fibrosis (CF), and is the developer of Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor), and Symdeko (tezacaftor/ivacaftor combination therapy), three approved CF therapies.
The new partnership will lean on Ribometrix’s proprietary discovery platform for identifying and optimizing small molecules that modulate the function of RNA, and on Vertex’s scientific, clinical, and regulatory capabilities. Ribometrix will receive a $20 million upfront payment from Vertex, including an equity investment.
RNA is a blueprint copy of DNA, essential to convert messages contained in genes into proteins. Many RNAs implicated in disease contain three-dimensional “pockets” (regions) that can be targeted by small molecule ligands.
Ribometrix’s platform uses multiple technologies to identify these three-dimensional domains in clinically relevant RNAs, and then discover small molecules that bind to them in ways that can lead to therapeutic benefits.
According to Ribometrix, potential advantages of this new class of investigational medicines over other protein and RNA-based approaches is that it can have a more potent and selective therapeutic effect, and a better tissue distribution. Additionally, it could allow penetration into the central nervous system, and be taken orally.
“Targeting RNA is a potentially powerful new approach for developing small molecule therapeutics, and Ribometrix has developed an innovative technology for the accelerated discovery of selective candidates in this new space,” Mark Bunnage, Vertex’s senior vice president and site head of Boston Research at Vertex, said in a press release.
“This partnership adds a novel mechanism to our internal toolkit which has the potential to enhance our ongoing research capabilities, and support the expansion of our pipeline of transformative medicines in serious diseases,” Bunnage said.
The collaboration covers up to three therapeutic discovery programs, although the companies didn’t disclose information about them.
“As a company founded on groundbreaking research and expertise in RNA structural biology, we have seen firsthand how new technologies are dramatically expanding the druggability of RNA, and the potential for transforming the treatment of previously intractable diseases,” said Michael Solomon, PhD, Ribometrix’s CEO.
“Vertex is a leader in creating transformative medicines through serial innovation, and we are proud to partner with them to rapidly and effectively develop a core discovery platform of small molecule therapeutics targeting RNA,” Solomon said.