The Cystic Fibrosis Foundation is funding the development of a gene editing therapy that is designed to use ReCode’s tissue-specific delivery vehicle to transport gene editing cargo to the lung cells in people with cystic fibrosis.
RT’s Three Key Takeaways:
- Cystic Fibrosis Foundation backs gene editing: The foundation is investing up to $15 million in ReCode Therapeutics to support a collaboration with Intellia Therapeutics aimed at developing a gene editing therapy for cystic fibrosis.
- Focus on lung-targeted therapy: The funding will help optimize ReCode’s lipid nanoparticles to deliver gene editing therapies directly to lung stem cells, which are critical for repairing damaged lung tissue in CF patients.
- Addressing unmet needs in CF: The project aims to develop therapies for patients with rare CF mutations who are not eligible for existing CFTR modulators, with potential long-term plans for systemic treatment to target other affected organs.
The Cystic Fibrosis Foundation has agreed to invest up to $15 million in ReCode Therapeutics to support its gene editing collaboration with Intellia Therapeutics.
The funding is intended to be used to develop a gene editing therapy that could be delivered into the lung cells of people with cystic fibrosis (CF).
ReCode is focused on developing a version of its lipid nanoparticles that is optimized for delivery to lung stem cells, the ideal target for a permanent gene editing therapy. Similar to a pill capsule that protects medicines, lipid nanoparticles shield and direct genetic material to cells without damaging the material along the way. Lung stem cells are important because they create new lung cells to replace damaged or dying ones. Delivering genetic-based therapies to specific cells in the lungs remains a significant challenge in medicine.
Targeting the Lungs for Gene Correction
While ReCode concentrates on the delivery system, Intellia is working to develop the gene editing therapy being delivered. Intellia’s therapy is designed to target permanent or near-permanent correction of a specific nonsense mutation (also called a “stop” mutation) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures.
“Gene editing is a promising, complex technology, which will take many years to develop into a therapy for people with CF,” says Steven Rowe, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation, in a release. “By investing in this research now, we are laying the foundation to develop what we hope will be a transformative treatment for people with CF, especially those who can’t take CFTR modulators.”
The companies are initially targeting the lungs for gene correction because CF severely impacts the lungs and can cause respiratory failure—a leading cause of death in people with the disease. Eventually, a systemic treatment will need to be developed to correct CF in other organs impacted by the disease, such as the pancreas.
Additional Investments in CF Therapies
The funding for the gene editing program is in addition to a separate $15 million investment in ReCode that the foundation made to develop a messenger RNA therapy, which is being tested in phase 1 and 2 clinical trials. Messenger RNA therapy is another potential treatment that could help all people with CF, especially those who aren’t eligible for or cannot tolerate CFTR modulators.
“We are grateful to the CF Foundation for their ongoing support of our efforts to develop novel treatments for people living with cystic fibrosis,” says Shehnaaz Suliman, MD, MBA, MPhil, chief executive officer of ReCode Therapeutics, in a release. “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.”
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