CF Foundation invests $3M in experimental therapy RCT2100
Organization has put a total of $33M into Recode, the drug's developer
The Cystic Fibrosis Foundation has made an additional $3 million investment in Recode Therapeutics to support clinical development of RCT2100, an investigational therapy for cystic fibrosis (CF) that is currently being tested in patients who cannot take CFTR modulators.
The CF Foundation had previously invested $15 million in Recode to support the development of and early-stage clinical trials for RCT2100, as well as $15 million to support the development of gene-editing therapies for CF. All told, the foundation has now invested $33 million in the company, which just announced more than $29 million in additional financing to advance its pipeline of investigational therapies for CF and other disorders.
“With continued support from organizations like the CF Foundation … we are building on our momentum to deliver on the promise of genetic medicines for people living with genetic diseases who currently have limited or no effective treatments,” Shehnaaz Suliman, MD, Recode’s CEO, said in a company press release.
Clincal trial currently testing RCT2100
CF is caused by mutations in a gene called CFTR, which contains instructions to make a protein of the same name that is key to the regulation of mucus production. People with CF have no or faulty CFTR protein, leading to the production of thick, sticky mucus that accumulates in the lungs and other organs, driving most disease symptoms.
CFTR modulators are a class of medications that can increase CFTR protein functionality in people with specific disease-causing mutations. But some patients can’t benefit from modulator therapy because they don’t have responsive mutations, while others are unable to take modulators due to side effects.
Protein production starts by copying genetic code in a cell’s DNA into messenger RNA (mRNA), a temporary molecule that is used as a template to make proteins. RCT2100 is an inhaled therapy designed to deliver mRNA that encodes a healthy version of the CFTR protein to lung cells, allowing cells to produce a functional protein. Since this approach is not dependent on a patient’s underlying mutation, it’s expected to work similarly in all CF patients, according to Recode.
A Phase 1b clinical trial (NCT06237335) of RCT2100 is ongoing. The first part of the trial is evaluating the therapy’s safety in healthy volunteers, and the second part is assessing it in adults with CF who cannot take CFTR modulators or do not use them due to intolerance. The trial is enrolling participants at sites in the U.S., U.K., France, and the Netherlands.
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