Gene Therapy: What To Expect Now And In The Future
A Look At The State Of The Industry
The US FDA could approve as many new gene therapies in 2023 as it has in the previous five years. Development in the field has rapidly accelerated, but still faces hurdles.
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BioMarin, Sarepta and bluebird bio are all launching new gene therapies in the US. The drug makers updated investors on the commercial execution during second quarter financial earnings calls.
Chief development officer William Pao oversees Pfizer’s clinical pipeline. He joined the company from Roche following a career in academia, and after his first year at Pfizer talked to Scrip about the transition to late-stage drug development.
BioMarin has finally brought its hemophilia A gene therapy Roctavian market and is determined prove naysayers wrong, but must work with its limitations and a confusing US label.