Manufacturing problems with other new medicines highlight difficulty of developing advanced treatments
When it comes to gene therapies, it seems anything that can go wrong, will go wrong. A month after its new gene therapy drug Zolgensma (onasemnogene abeparvovec-xioi) was approved for use, Novartis told regulators that some of its preclinical data had been tampered with. Meanwhile, other gene therapy companies have their own headaches to deal with.