Mainstreaming cell and gene therapy – Realizing its potential

BioPharma Dive November 28, 2022
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Mainstreaming cell and gene therapy clinical trials

Cell and gene therapies (CGT) are starting to live up to their promise, not only to create treatments for previously untreatable diseases but also, in some cases, to provide actual cures. The first cell therapy, Novartis’ Kymriah, a CAR-T treatment, was approved¹ in 2017 for relapsing and recurring diffuse large B-cell lymphoma, but it has added additional indications. The same year, the first gene therapy, Spark Therapeutics’ Luxturna, was approved² to treat retinal dystrophy caused by biallelic mutations in the RPE65 gene. The mRNA COVID-19 vaccines developed by Pfizer-BioNTech and Moderna also fall into the category of CGT.

This article looks at the CGT landscape, the challenges biopharma companies face in running...

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