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Nonprofit issues challenge to accelerate treatments for every person with CF
After watching the second plenary of the 2021 North American Cystic Fibrosis Conference, I was incredibly impressed by the level of support the Cystic Fibrosis Foundation is providing to the development of the next generation of therapeutics. Developing genetic therapies -- especially those as complex as gene editing -- will take a long time and a lot of collaboration.
The Cystic Fibrosis Foundation has awarded up to $3 million to Kinnear Pharmaceuticals to conduct preclinical testing of a broad-spectrum anti-infective that has the potential to treat multi-drug resistant Pseudomonas and other infections in people with cystic fibrosis.
Delivery of genetic therapies to affected tissues is a key challenge to developing new treatments for people with cystic fibrosis. In addition to investing in viral delivery and lipid nanoparticles, the Cystic Fibrosis Foundation is looking at the potential of a relatively new approach to delivery using an exceptionally small synthetic molecule called a polymer nanoparticle.
Funding will support preclinical research and early-stage clinical trials
The Cystic Fibrosis Foundation’s investment will go toward conducting preclinical research on a novel gene delivery vehicle. If successful, this gene delivery method could overcome some of the biggest challenges to delivering a gene therapy into the lung cells of people with cystic fibrosis.
Today, the Cystic Fibrosis Foundation announced a collaboration with Deep Science Ventures to accelerate treatments that address the underlying cause of CF for every person with the disease. The collaboration will focus on uncovering and designing new technologies to address key scientific challenges on the Path to a Cure.
As someone who does not qualify for Trikafta®, I took particular interest in the first plenary of the North American Cystic Fibrosis Conference, which discussed potential treatments for people with nonsense and rare mutations. I learned that researchers are exploring a variety of options, and some early research has shown promising results.
The Cystic Fibrosis Foundation is providing up to $5 million to develop a method to deliver a healthy copy of the CFTR gene into the lung cells of people with CF that is unlikely to trigger an immune system response.