by Irena Maragkou – Pharmaceutical Technology –
Innovative cell and gene therapies (CGTs) have significantly disrupted the treatment landscape for several genetic diseases. In recent years, the number of CGT approvals has been on an upward trajectory, with there being ten global approvals announced just last year.
According to a survey completed by GlobalData in November 2023, CGT was scored as the industry trend to have the greatest impact on the pharmaceutical industry in 2024, followed by personalised/precision medicine.
Currently, there are 69 marketed CGTs worldwide, approved by regulatory bodies such as the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and others. In 2023, a total of seven novel CGTs received FDA approval including notable treatments like Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), and bluebird bio’s Lyfgenia (lovotibeglogene autotemcel). The acceleration in approvals in recent years not only highlights the staggering potential of these therapies but also the growing demand for specialized and expanded manufacturing capabilities.