Opinion: Developing a new gene therapy was hard. So was figuring out how to get it to patients
After a career spent in pharmaceutical commercial strategy and operations, I thought I had a handle on what was needed to launch a new therapy. A new gene therapy taught…
After a career spent in pharmaceutical commercial strategy and operations, I thought I had a handle on what was required to launch a new therapy. But I’ve learned many lessons — and faced a few curveballs — in the 12 months since the FDA approved Luxturna (voretigene neparvovec), a one-time gene therapy for the treatment of patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
To bring this first-of-its-kind therapy to U.S. patients, Spark Therapeutics, where I serve as chief commercial officer, had
