Data manipulation casts shadows on Novartis gene therapy

A scientific illustration showing an adeno-associated virus

Source: © Science Photo Library

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.