Modern pharma is increasingly about the applying real-world evidence to gene therapies

Transformative gene therapy is redefining how we treat certain diseases but – as with any innovative approach – there are challenges afoot.

In many cases, gene therapies are being introduced for the treatment of rare monogenic diseases – conditions caused by a singular faulty or missing gene. There is potential to treat these diseases effectively with a one-time dose, but approval processes for therapies need to meet regulatory standards.

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