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What will be the approval status of the new small molecule pill for sickle cell disease by the end of 2025?
2
Resolution / Starting Odds
Approved in the US only • 25%
Approved in the US and EU • 25%
Approved in the US, EU, and other major markets • 25%
Not approved in any major market • 25%
Official announcements from FDA, EMA, and other regulatory bodies
Story
New Small Molecule Pill Offers Safer Treatment for Sickle Cell Disease, Published in Science Magazine
Jul 4, 2024, 06:20 PM
A groundbreaking discovery in the treatment of sickle cell disease has been published in Science Magazine. Researchers have developed a small molecule pill that acts as an epigenetic modifier to inhibit fetal hemoglobin, offering a less dangerous and potentially more accessible alternative to CRISPR genome editing, such as Casgevy. This new approach could open up treatment options globally, reducing the need for toxic conditioning. The study, led by a team including Pamela Ting and Jay Bradner, marks a significant advancement in chemical biology and the development of molecular glue degraders.
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Yes • 50%
No • 50%
Yes • 50%
No • 50%
Yes • 50%
No • 50%
Successful • 25%
Partially Successful • 25%
Unsuccessful • 25%
No Data Released • 25%
Yes • 50%
No • 50%
Approved in USA only • 25%
Approved in EU only • 25%
Approved in both USA and EU • 25%
Not approved in USA or EU • 25%
Accelerated Approval • 25%
Full Approval • 25%
Conditional Approval • 25%
No Approval • 25%
Yes • 50%
No • 50%
