Tiny Biotech Takes on Goliath Glaxo's $730,000 Gene Therapy

(Bloomberg) -- GlaxoSmithKline Plc has seen precious few takers for its $730,000 gene therapy for a rare and life-threatening immune illness known as “bubble-boy disease.” Now a tiny startup thinks it can succeed where the U.K.-based giant is struggling.

In almost two years since Glaxo's Strimvelis came to the market, just five patients have received the treatment, offered only through a single center in Milan. Orchard Therapeutics Ltd., a closely held company in London with about 80 employees and no products on the market, is preparing to sell a competing version that may reach more sick children.

Orchard, whose team includes three former Glaxo executives, wants to one-up the larger company by taking aim at a Strimvelis shortcoming. While Glaxo's therapy can't be transported, Orchard is planning to use frozen cells that can travel to markets all over the world.

“Having patients move to one center in another country is not an easy way to manage things,” Orchard Chief Executive Mark Rothera said in an interview. His company's approach “means patients can be treated where they live.”

Gene therapy offers the possibility of using a single procedure to cure DNA flaws that cause severe, sometimes fatal conditions. But some developers are examining their commitment to the field, facing questions about the therapies' profitability and prices, which have ranged up to $1 million.

Glaxo was hailed as a pioneer for its work to develop Strimvelis, just the second gene therapy for an inherited disease to gain approval. Now CEO Emma Walmsley is pruning likely duds from the company's pipeline of drugs and is seeking a partner for its rare disease unit, which developed Strimvelis.

Orchard's therapy, called OTL-101, is designed to be frozen, or cryopreserved, so that it can be sent to patients anywhere. The startup recently raised $110 million, plans to double its staff to about 200 by the end of this year, and is considering a sale of stock, Rothera said. Partnering with Glaxo is a possibility, he said. 

“We could be a potential party that would be of interest,” said Rothera, who was chief commercial officer of biotech PTC Therapeutics Inc. before joining Orchard last year. “GSK has to make their decision. I'm sure they've had a lot of interest in this.”

Harmless Virus

Glaxo declined to comment on potential collaborators, saying it's “focused on finding the right partner who can ensure these medicines get the focus they deserve.”

Bubble-boy disease disables children's immune systems and takes its nickname from a patient who spent almost all of his life in a plastic chamber made to protect him from infections. The condition is called severe combined immune deficiency, and Glaxo's product treats a form resulting from defects in a gene that makes a key immune enzyme. Donors for stem-cell transplants, a standard treatment, can be hard to find, and weekly injections of a replacement enzyme tend to lose their effectiveness.

Strimvelis uses a harmless virus to insert a healthy copy of the defective gene into the bone marrow where immune cells are produced. Only 15 new cases are diagnosed annually in Europe, and Glaxo has said their rarity explains the low number of patients treated. Worldwide, there may be 100 to 200 babies born in developed nations each year with the disease, Rothera said.

More than 50 patients have been treated with Orchard's OTL-101 and all are still living. The company expects to file for U.S. approval this year and begin selling the treatment in 2019, seeing it as a launchpad to a wider pipeline of gene therapies, Rothera said.

Glaxo said it has also long been interested in freezing cells to ship Strimvelis to far-flung sites. Cryopreservation increases cells' maximum six-hour shelf life to several weeks and potentially months, according to Glaxo.

“So far there's no option of this nature in the U.S., there's no option in Latin America, and no option in Asia,” Rothera said. “We want to build on GSK's heritage and the foundation they've already established.”

To contact the reporter on this story: James Paton in London at jpaton4@bloomberg.net.

To contact the editors responsible for this story: Eric Pfanner at epfanner1@bloomberg.net, John Lauerman

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