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How many CF patients will receive the new prime editing gene therapy within the first year of its approval?
2
Resolution / Starting Odds
Less than 1,000 • 25%
1,000 to 5,000 • 25%
5,000 to 10,000 • 25%
More than 10,000 • 25%
Healthcare and medical records, official treatment reports
Story
New Gene Editing Advances Show Promise for Treating CFTR F508del Mutation in Cystic Fibrosis
Jul 10, 2024, 12:02 PM
Recent advancements in gene editing have shown promising results in treating cystic fibrosis (CF). Scientists have developed a prime editing approach that efficiently corrects the most common CF mutation, CFTR F508del, in primary airway cells from CF patients. This method could pave the way for a one-time treatment as effective as existing daily therapies. Additionally, new lipid nanoparticle designs have enabled targeted in vivo lung editing in mice, potentially leading to new treatment options for CF. Researchers are optimistic about the potential of these developments to offer a permanent solution for CF patients.
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Yes • 50%
No • 50%
Phase 1 • 25%
Phase 2 • 25%
Phase 3 • 25%
No clinical trials • 25%
Pfizer • 25%
Moderna • 25%
Novartis • 25%
Other • 25%
Less than 1,000 • 25%
1,000 to 5,000 • 25%
5,000 to 10,000 • 25%
More than 10,000 • 25%
Yes • 50%
No • 50%
Less than 50 • 25%
50-100 • 25%
101-200 • 25%
More than 200 • 25%
USA • 25%
UK • 25%
Germany • 25%
Other • 25%
Less than 500 • 25%
500-1000 • 25%
1001-2000 • 25%
More than 2000 • 25%
Yes • 50%
No • 50%
None • 25%
One • 25%
Two • 25%
Three or more • 25%