Gene Replacement
FA212
FA212/UPenn
Pre-clinical
FA212 is a gene replacement therapy with dual delivery to both the heart and the dentate nucleus of the brain using an AAV9 variant for delivery.
HOW DOES A DRUG GET DEVELOPED?
Stages of Development for FA212
The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.
In mouse models, FA212 shows efficacious transduction of the heart and the dentate nucleus at low doses that avoid DRG toxicity. The company is completing preclinical studies.