Bubble boy hope: Strimvelis gene therapy revises genetic make-up, offers rare immune deficiency cure

Autosomal recessive II

A child in Europe has become the second individual ever to receive a commercial gene therapy, according to GlaxoSmithKline.

The treatment, called Strimvelis, can provide an outright cure for a rare inherited immune deficiency by revising a patient’s genetic makeup (see “Gene Therapy’s First Out-and-Out Cure Is Here”).

Gene therapy has been widely explored in experimental medical studies, but its commercial potential is largely untested…

GlaxoSmithKline spokesperson Anna Padula said the company treated its first patient in March, nearly a year after Strimvelis was approved for sale in Europe in May 2016.

Gene therapies are more complex than ordinary pills, and the diseases they treat affect few people. But sky-high prices have raised questions about whether patients can access them. Strimvelis, used to treat an ultra-rare immune deficiency, has a list price of 594,000 euros, or $648,000, making it one of the most expensive drugs available (see “Gene-Therapy Cure Has Money-Back Guarantee”).

Strimvelis treats a rare disease called severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, which leaves babies without a fully functioning immune system and vulnerable to infections.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: A Year After Approval, Gene-Therapy Cure Gets Its First Customer

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