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.
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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.
Last week I travelled to D.C. to serve on a panel discussing the recent developments in precision medicine. I've got to say, it was pretty neat.
My experience surviving a life-threatening infection led to my role as the CF community co-chair of the Cystic Fibrosis Foundation's Infection Research Initiative. Having a room full of leading scientists listen to my story helped renew my faith that we can tackle the complex challenge of difficult-to-treat infections.
The Cystic Fibrosis Foundation awarded up to $5 million to Armata Pharmaceuticals for the first-ever controlled clinical study of phage therapy in CF, reaffirming the Foundation's commitment to advance innovative solutions to the growing challenge of antibiotic resistance.
The Cystic Fibrosis Foundation has awarded up to $5.6 million to Microbion Corporation to develop a novel, inhaled antibiotic to treat drug-resistant bacterial infections in people with cystic fibrosis.
Thirty-three new projects are being funded as part of the Foundation's $100 million Infection Research Initiative.
Although 90-95 percent of people with cystic fibrosis are expected to benefit from CF transmembrane regulator (CFTR) modulators, 5 percent of the population will still need alternative therapies to address the underlying cause of their disease. Learn more about the research that the Cystic Fibrosis Foundation is doing to find treatments for the 5 percent of people with these rare and nonsense mutations.
This year, theratyping, the process of matching medications with mutations, enabled more people with cystic fibrosis who have rare mutations to benefit from modulators. In the first plenary at the NACFC, we heard more about how theratyping will be used, as well as other future advances coming in CF treatment.
The Cystic Fibrosis Foundation has entered into an agreement with Synspira Therapeutics Inc. to develop a non-porcine enzyme replacement therapy to offer an alternative to people with cystic fibrosis who cannot digest food properly.