Page Title
Drug Development Pipeline
Prime Medicine
Status
Pre-clinicalTherapeutic Approach
Genetic Therapy
This program is developing an approach called prime editing, a type of gene editing that has the potential to correct many different types of CF-causing mutations. Prime editing involves inserting or deleting small pieces of DNA at precise locations in a gene.
This program is studying two different technologies using use prime editing to fix the CFTR gene. The first, called “hotspot,” uses prime editing to make smaller corrections to specific CFTR mutations. Prime Medicine has already begun to use hotspot to correct the G542X nonsense CFTR mutation in the lab and will extend this work to develop therapies for other CFTR mutations. The second, called PASSIGETM, uses prime editing to make large gene insertions, which could potentially work in nearly any person with CF, regardless of their underlying CFTR mutations.
Status
Laboratory studies to develop and test these technologies are underway.
Sponsor
This program is sponsored by Prime Medicine and partially funded by the Cystic Fibrosis Foundation.

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