Cystic Fibrosis Foundation Commits Up to an Additional $24 Million for Prime Medicine to Develop Gene Editing Therapy
Prime Medicine uses a gene editing technology called prime editing — a technology that enables a wide range of modifications to the DNA with a high degree of precision. The company — founded by Drs. David Liu and Andrew Anzalone, who pioneered the development of this unique editing technology — is investigating whether prime editing could treat several diseases, including CF.
The CF Foundation's initial investment in Prime Medicine supported work to demonstrate the versatility of prime editing to correct multiple CF-causing mutations in the lab. This additional investment by the CF Foundation will focus on the development of a prime editing therapy targeting the nonsense mutation G542X — one of the most prevalent CF-causing nonsense mutations and one for which there are no available therapies.
'We believe gene editing offers the best hope for a cure for cystic fibrosis because it could permanently correct the mutations that cause this disease,' said Michael P. Boyle, MD, president and chief executive officer of the Cystic Fibrosis Foundation. 'Our investment will help support gene editing research for CF through early development stages, including efforts to overcome the challenges of delivery to the lungs.'
As part of its ongoing efforts to develop a prime editing therapy, Prime Medicine is investigating the best way to deliver its gene editing treatment to the lungs of people with CF, a major challenge because of the thick, sticky mucus that clogs the airways and the body's natural immune response to block foreign entities from entering cells. If successful, the gene editing cargo will enter the lung cells that normally make CFTR protein and correct the CFTR mutation in the cell's DNA, resulting in a potentially permanent fix to the CFTR gene.
With continued advancement in prime editing and delivery to the lungs, this technology may eventually be used to correct many types of CF mutations. Prime Medicine is advancing multiple approaches in CF, including 'hotspot,' which uses prime editing to make smaller corrections to specific CFTR mutations and PASSIGE, which uses prime editing to make large gene insertions. These combined strategies eliminate the need to develop new prime editing therapies for each individual mutation.
Previously, the Foundation committed up to $15 million to Prime Medicine to pursue development of their gene editing technology for CF.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.
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