CF Foundation invests up to $15M in Prime gene-editing technology
Funding aims to accelerate development of 2 Prime Editing strategies
The Cystic Fibrosis Foundation has announced new funding of up to $15 million to Prime Medicine to further develop its gene-editing technology — called Prime Editing — for cystic fibrosis (CF).
The award is part of the foundation’s $500 million Path to a Cure initiative, which aims to accelerate the preclinical development of novel treatments that address the underlying causes of CF.
“Our investment in Prime Medicine represents one of the Foundation’s key strategies to achieving a genetic therapy in cystic fibrosis: de-risking early-stage science to pave the way for the ultimate cure,” Steven M. Rowe, MD, the CF Foundation’s executive vice president and chief scientific officer, said in a press release, noting that “a genetic therapy for cystic fibrosis will take time to develop.”
CF is caused by mutations in the CFTR gene that lead to the malfunction or complete absence of a protein of the same name. These mutations result in the production of thick mucus in several organs, such as the lungs and the pancreas.
Prime Editing is a gene-editing technology that’s intended to fix the damaged CFTR gene. It works like a “DNA word processor, with the power to search and replace genetic sequences,” the company states on its website. According to the press release, Prime Editing may enable several types of CFTR mutations to be corrected with a single type of therapy.
Gene-editing technology could potentially be used for any CF patient
Specifically, the new funds will help accelerate the development of two of Prime’s platform technologies, according to a separate press release from Prime.
The first, Prime Assisted Site Specific Integrase Gene Editing — dubbed hotspot — makes small corrections in the CFTR gene. The company says this strategy is being developed to correct G542X, a common class 1 mutation, or one impacting protein production, that makes CFTR short, unstable, and rapidly degradable.
The second technology, called PASSIGE, for Prime Assisted Site Specific Integrase Gene Editing, uses Prime Editing to insert large portions of a gene. This strategy could potentially help nearly any person with CF, regardless of the type of mutation, according to the company.
Two main barriers complicate the delivery of DNA to CF lung cells: the thick mucus that builds up in cystic fibrosis that makes cells more difficult to reach, and getting through the cell membrane. To improve gene delivery to the lungs, Prime Medicine uses lipid (fat) nanoparticles, small and dense particles that are able to fuse with the cell membrane.
We are pleased to advance this work together with the CF Foundation, an organization devoted to delivering transformative therapies. … We believe their many resources and learnings will allow us to accelerate our ongoing efforts.
“Leveraging our Prime Editing technology, we hope to create a one-time, non-viral therapy, which can precisely correct the underlying genetic mutation that causes CF and potentially offer the first cure for this progressive, devastating disease,” said Keith Gottesdiener, MD, the company’s president and CEO.
“We are pleased to advance this work together with the CF Foundation, an organization devoted to delivering transformative therapies. … We believe their many resources and learnings will allow us to accelerate our ongoing efforts,” Gottesdiener added.
According to Prime, the CF Foundation will provide $6 million upfront. An additional $6 million will be awarded upon Prime achieving certain preclinical milestones. As much as $3 million in supplementary funding would be available upon mutual agreement by the parties.
The CF Foundation, for its part, sees this as “an exciting time for genetic technologies,” Rowe said.
“Last month marked the first FDA approval of a gene editing therapy for sickle cell disease, showing the potential of gene editing outside the lab. We want to bring that success to cystic fibrosis, so that all people with CF may benefit from a transformative treatment,” Rowe said.
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