MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, announced today that it will receive new funding from the Cystic Fibrosis Foundation (CF Foundation) to support ReCode’s gene correction research program with the goal of developing and commercializing new treatments for people with cystic fibrosis (CF), including those with genotypes that do not respond to, or are intolerant to, approved CFTR modulators. The gene correction program is being developed with Intellia Therapeutics, utilizing Intellia’s proprietary CRISPR-based gene editing platform, including its DNA-writing technology.
“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, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “This 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. 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.”
Key Highlights:
- The CF Foundation has agreed to invest up to $15 million in new funding to support gene correction therapeutic research, which is currently underway as part of a collaboration agreement with Intellia Therapeutics announced earlier this year. The programs leverage Intellia’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s next-generation Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform with the aim to correct the underlying genetic cause of CF and offer a potentially transformative therapy.
- The funding from the CF Foundation will support development activities for up to three different gene correction programs. These programs will initially focus on developing novel therapies for CF mutations that are not addressed or adequately treated by existing therapies.
- The funding is in addition to the CF Foundation's prior investment in ReCode to support the company’s ongoing RCT2100 clinical program investigational inhaled mRNA therapy, which is currently enrolling a Phase 1b multi-dose study in people with CF.
About Cystic Fibrosis
Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the CF Foundation. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs and other organs. It also results in a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of people in the CF community have genetic mutations that do not enable them to benefit from these life-changing therapeutics.
About ReCode Therapeutics
ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency.
ReCode’s lead programs include RCT2100 for the treatment of the 10% of people with cystic fibrosis who have genetic mutations in the CFTR gene that do not respond to currently approved CFTR modulators and RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including central nervous system, liver, and infectious disease indications.
ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram.
