New curative treatment for AATD in the pipeline
Prime Editing
Dear readers,
Good news!
A new company, called Prime Medicine, announced a curative treatment for Alpha-1 Antitrypsin Deficiency (AATD), especially for those with the Pi*Z mutation. Their approach is based on DNA editing (not RNA). That means it permanently corrects the genetic mutation in liver cells, instead of temporarily adjusting protein levels like RNA therapies.
It uses a highly precise method called Prime Editing, which works like a 'search and replace' tool to fix the mutation in your DNA without cutting the gene in half. The first human trials are expected around 2027, and this is a very promising potential cure.
What is Being Developed?
Permanently correction of the SERPINA1 gene mutation (Pi*Z) using Prime Editing, which directly edits your DNA using a molecular 'word processor' in liver cells.
This approach fixes the root cause of the disease instead of just managing the symptoms.
What is Prime Editing?
The type of editing is DNA editing (not RNA), which finds the exact mutation and changes it back to the healthy version. During early studies it showed no off-target changes, which means that it is considered safe and accurate.
What Have They Achieved So Far?
In mouse models with human-like mutations, it corrected up to 72% of liver cells and restored Alpha-1 protein levels to normal. More than 95% of the protein made was the healthy M-form.
In monkeys, more than 50% editing efficiency was achieved with no detectable harmful side effects
Why This Is Important
Prime Editing offers a potential one-time cure, which is promising for both lung and liver.
What’s next?
Final Thoughts
This is again an exciting development in AATD therapy. While still a few years away, Prime Editing provides hope for a genetic cure, and it is based on a safer and precise gene editing technology.
We will continue to monitor this company's developments and keep you informed!
