Promising pipeline for cell and gene therapies

By Ben Hargreaves

- Last updated on GMT

(Image: Getty/Vchal)
(Image: Getty/Vchal)

Related tags Novartis Gilead bluebird bio

With nearly 300 cell and gene therapies in development, targeting more than 100 diseases, 2019 could be set to be a big year for cell and gene therapies.

The rapid development of cell and gene therapies has been spurred on by growing investment from both industry​ and from national governments​. The investment has seen the arrival of chimeric antigen receptor (CAR)-T treatments​, and the first approval for a gene therapy​ to treat a genetic disease.

A recent report​ by PhRMA, an association representing the pharmaceutical industry in the US, outlines the number of cell and gene therapies in the clinic, and in what particular disease areas the focus is:

In total, there are 289 cell and gene therapies in various stages of clinical development and the candidates cover more than 100 diseases, with nearly half of these candidates within oncology.

“These concepts are no longer the stuff of science fiction, but rather, real-life science where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; where new genes can be introduced into the body to combat disease; and where adult stem cells can generate replacements for cells that are lost to injury or illness”​, said Scott Gottlieb, commissioner of the US Food and Drug Administration, in a statement regarding streamlining the regulatory paths for cell and gene therapies.

Alongside approvals for CAR-T therapies, there were also US and European approvals for Spark Therapeutics gene therapy, Luxturna (voretigene neparvovec-rzyl), which Novartis has licensed for ex-US sales​. Alnylam also secured the first ever approval for an RNAi therapeutic​.

With 2018 being a busy year for cell and gene therapy approvals, taking the total number of such medicines approved to five, the amount of treatments in late-stage trials should see this increase in 2019 – with bluebird bio, alone, holding three gene therapy treatments close to commercialisation​.

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