Can Cell and Gene Therapies Achieve Their Full Potential?
Cell and gene therapies (C>s) have made huge advances in a few short decades. By February 2020, five therapies were on the market and FDA had approved nine more, while 362 treatments were in the US pipeline, up 25% from the previous year, according to the Pharmaceutical Research and Manufacturers of America’s latest survey (1). In September 2020, the first patient was dosed with autologous chimeric antigen receptor (CAR-T) lymphoma treatment using an automated closed system. “Despite complex and mainly manual processes, we can produce these therapies. Now we must work on making processes reproducible,” says Joerg Ahlgrimm, president and chief operating officer at The Discovery Labs, who was previously head of global C> operations at Lonza Pharma & Biotech. Significant progress has already been made, he notes. “The fact that we can routinely upscale a viral vector to 200–500 L standard represents a huge accomplishment. Years ago, we could not even have imagined reaching this point. And now, we are approaching 2000-L batch sizes,” he adds…