Gene Therapy’s First Out-and-Out Cure Is Here.


Interesting idea...wonder if it is on track for approval here in the USA too?
Full text here.

Strimvelis uses a “repair and replace” strategy, so called because doctors first remove stem cells from a patient’s bone marrow then soak them with viruses to transfer a correct copy of the ADA gene.

“What we are talking about is ex vivo gene therapy—you pull out the cells, correct them in test tube, and put the cells back,” says Maria-Grazia Roncarolo, a pediatrician and scientist at Stanford University who led the original Milan experiments. “If you want to fix a disease for life, you need to put the gene in the stem cells.”
Some companies are trying to add corrected genes using direct injections into muscles or the eye. But the repair-and-replace strategy may have the larger impact. As soon as next year, companies like Novartis and Juno Therapeutics may seek approval for cancer treatments that also use a combination of gene and cell therapy to obliterate one type of leukemia.

Overall, investment in gene therapy is booming. The Alliance for Regenerative Medicine says that globally, in 2015, public and private companies raised $10 billion, and about 70 treatments are in late-stage testing.
GSK has never sold a product so drastically different from a bottle of pills. And because ADA-SCID is one of the rarest diseases on Earth, Strimvelis won’t be a blockbuster. GSK estimates there are only about 14 cases a year in Europe, and 12 in the U.S.

Instead, the British company hopes to master gene-therapy technology, including virus manufacturing. “If we can first make products that change lives, then we can develop them into things that affect more people,” says Kili. “We believe gene therapy is an area of important future growth; we don’t want to rush or cut corners.”


This is the very first time I've heard of a drug manufacturer considering offering a money back guarantee!

From Wikipedia:

The price for the treatment was unknown as of April 2016, but a comparable treatment is Glybera, the first gene therapy approved by European regulators in 2012, which costs about $1m per patient.[7] GSK said that it was considering payment models, including payments made over several years, and a risk-sharing program where payers get a refund if the treatment doesn't work.[8

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