Primera is dedicated to advancing its programs to be first into the clinic that will apply gene editing technologies directly to mitochondrial mutations that drive mitochondrial disease and potentially broader disease targets that have mitochondrial dysfunction as a component.
Pipeline: Multiple Mitochondrially Driven Disorders
Expansion Into Other Diseases
with he m.3243A>G Mutation
MELAS is a systemic disease not necessarily included in these numbers represents an additional 87K patients.
As other editors come online, more patients will become available.