Why It’s the Best Time to Be in Cell and Gene Therapy

Cell and gene therapies are here, and they’re here to stay. We stopped to chat about the future of the field with Alexander Vos last week at Bio Europe Spring.

The cell and gene therapy space has been given a big boost in the last couple of years. Since 2016, GSK’s gene therapy Strimvelis is available to children with a rare disease known as ADA-SCID. Last year, the first two CAR-T cell therapies for cancer were approved in the US, changing the way certain blood cancers are treated. And since December, Spark’s Luxturna can be used in the US — and soon in Europe — to fix a genetic mutation that causes blindness.

“With some many recent approvals, it’s never been a better time for the cell and gene therapy space,” says Alexander Vos, an expert in the field as the former CEO of PharmaCell, a Dutch cell therapy company acquired last year by Lonza.

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“For certain types of patients, really curative therapies have been developed for the very first time,” says Vos. Indeed, Strimvelis and Luxturna can treat diseases where no other treatment is available, while CAR-T cells have been able to cure some cancer patients for which all other available therapies had failed.

These breakthroughs seem to be driving a wave of enthusiasm regarding what these treatments can do next. But it is important to remember the field is still quite young and some issues still need to be addressed.

One of them is pricing. These new cell and gene therapies have six-figure price tags. And although those figures can be lower than the long-term expenses of current forms of treatment, not all health insurance systems can easily sustain such a big one-off payment. The first gene therapy, uniQure’s Glybera, had to be withdrawn for precisely this reason.

As Vos points out, one way cell and gene companies are addressing this issue is through new reimbursement schemes based on the therapy’s risk. For example, GSK is offering a money-back guarantee of the full €594,000 tag of Strimvelis if the treatment does not work. Novartis is doing something similar with its CAR-T therapy Kymriah, charging only those patients that respond to the therapy within the first month.

Vos believes the next few years will see the prices go down considerably as the technology and the manufacturing processes are fine-tuned and automated and as they become available to a wider range of indications. Still, that reduction will have a limit. I don’t think we can expect the cost to ever be the same as for antibodies.”

When looking into the future, Vos is confident that cell and gene therapy will make big advances in the next 5 to 10 years for diseases with short-term mortality, such as cancer. However, for more complex, chronic diseases such as diabetes, he expects “it will take a long time to get there.”

With the upcoming European approval of CAR-T cells and Luxturna, and many other treatments in the pipeline, especially for rare diseases, now seems to be the time to be in cell and gene therapy.

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