CF Foundation invests in Recode to support gene editing for CF
Collaboration with Intellia Therapeutics would develop new treatment strategy
The Cystic Fibrosis Foundation is investing up to $15 million in Recode Therapeutics to help spur the development of new gene-editing medicines for people with cystic fibrosis (CF) who don’t have effective treatment options.
“We are grateful to the CF Foundation for their ongoing support of our efforts to develop novel treatments for people living with cystic fibrosis,” said Shehnaaz Suliman, MD, CEO of Recode, said in a company press release.
CF is caused by mutations in the CFTR gene, which provides instructions to make the CFTR protein. In CF, the lack of functional CFTR results in an unusually thick and sticky mucus being produced that builds up in organs, driving most of the disease’s symptoms.
A new class of medications called CFTR modulators has recently revolutionized care for most CF patients. Modulators can improve the functionality of the mutated CFTR protein, but only work in people with certain mutations.
Working in collaboration with Intellia Therapeutics, Recode is developing a new treatment strategy that would alter the genetic code in patients’ cells to create a healthy version of the CFTR gene, allowing functional protein production. Since this approach doesn’t target a patient’s mutated CFTR protein, it’s expected to work even in people who aren’t eligible for modulators.
The project would combine Intellia’s gene-editing technology with Recode’s tech to deliver the gene-editing machinery into patient cells. The funding will support development for up to three such gene-correction programs, according to Recode. The company didn’t name specific mutations that will be targeted, but said the project “will initially focus on developing novel therapies for CF mutations that are not addressed or adequately treated by existing therapies.”
Suliman said the new funding “will support our gene correction approach, enabling us to significantly accelerate research in collaboration with Intellia and to advance the development of a potentially transformative treatment for CF.”
Continuing investment in Recode
This funding marks the second major investment in Recode from the CF Foundation. The foundation previously invested up to $15 million to support Recode’s development of RCT2100, an experimental therapy currently in clinical testing.
Whereas the collaboration with Intellia focuses on gene editing to alter patient DNA, RCT2100 is an inhaled therapy that’s intended to deliver a healthy version of CFTR messenger RNA (mRNA) to lung cells. mRNA is an intermediary molecule made when the CFTR gene is read to make a protein. By delivering healthy CFTR mRNA, RCT2100 should allow the production of functional CFTR in lung cells. Like gene editing, the mRNA approach is expected to work equally well in all patients irrespective of their underlying mutation.
“Along with RCT2100, our inhaled mRNA program, we are building a therapeutic franchise with the goal of expanding disease-modifying treatment options for all people with CF,” Suliman said.
RCT2100 is being tested in a two-part Phase 1 clinical trial (NCT06237335). The first part is testing the therapy in healthy volunteers. The second part is testing it in people with CF and has started dosing patients. Recruitment in that study is ongoing in New Zealand and the Netherlands. Additional sites in the U.S., the U.K., and France are expected to enroll patients in the future.
About the Author
