Gene Replacement
ASP2016
Astellas Gene Therapies
Phase I
ASP2016 is an investigational AAV gene therapy designed to restore functional FXN expression and increase frataxin protein levels to restore normal cellular functions.
HOW DOES A DRUG GET DEVELOPED?
Stages of Development for Astellas ASP2016
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.
AT808, the gene therapy candidate obtained as part of its $3 billion acquisition of Audentes never made it through the preclinical stage. Astellas is dividing it into two separate programs to target two different aspects of Friedreich’s ataxia. ASP2016 is the first targeting cardiac aspects of FA.
Preclinical safety and efficacy studies supported clinical development of ASP2016.
April 2024: Astellas announced the FDA has cleared its IND for ASP2016 in April 2024. The company is hoping to dose the first patient in the second half of 2024, according to Richard Wilson, who leads genetic regulation at Astellas Gene Therapies.