The $1 Billion Bet on Pre-Symptomatic Medicine: Who Will Own the Longitudinal Baseline?
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Prime Medicine
Prime editing company developing genetic therapies for inherited diseases.
What they do
Prime Medicine is advancing PM647, an investigational in vivo prime-editing therapy for alpha-1 antitrypsin deficiency. The FDA cleared its investigational new drug application in September 2026, allowing a US clinical study of the approach targeting the PiZZ genotype. Clinical efficacy remains to be established.
The record
- Vertical
- Pharma & Drug Discovery
- Sub-segment
- Prime editing
- Product type
- Platform software
- Stage
- Public
- Headquarters
- Cambridge, MA, USA
- Region
- North America
- Founded
- 2019
- Total funding
- Undisclosed
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