EXOME

all the information, none of the junk | biotech • healthcare • life sciences

Fresh Off GSK Deal, Gene Therapy Startup Orchard Bags Another $150M

Xconomy Boston — 

Another gene therapy startup looks primed for an IPO. Orchard Therapeutics has just landed another $150 million to help drive forward a group of gene therapies it bought from British pharma company GlaxoSmithKline earlier this year.

The Series C funding round from Deerfield Management, RA Capital Management, Venrock, and others, includes a number of “crossover” investors that back both public and privately held companies, often a signal of a future IPO. And it’s the second big funding for Orchard, following a $110 million Series B round last year. The company has offices in London, Boston, and San Francisco.

The deal comes just four months after Orchard bought all of GSK’s (NYSE: GSK) gene therapy assets by handing the pharma giant a 19.9 percent stake in the privately held biotech. Those assets included Strimvelis, one of the few marketed gene therapies, approved in Europe in 2016 to treat a rare immune disease, adenosine deaminase severe combined immunodeficiency (ADA-SCID). But Orchard got experimental programs for metachromatic leukodystrophy (MLD), Wiskott-Aldrich syndrome (WAS), and beta thalassemia—all in human testing—in the deal as well, not to mention rights to three other preclinical gene therapies being developed for rare diseases by Fondazione Telethon and Ospedale San Raffaele.

Unlike conventional medicines, gene therapies are meant to provide a durable effect, if not a cure, with a single treatment. After years of ups and downs, gene therapies have seen scientific validation across several diseases. Although questions remain about how long gene therapies will last, whether safety problems will emerge, or whether they can be successful commercially, three products have been approved in Europe and the U.S., and more are likely on the way. Encouraging data continue to emerge for disease like hemophilia, Duchenne muscular dystrophy, spinal muscular atrophy, and more. Several gene therapy developers are now publicly traded.

There are two main types of gene therapy: an in vivo process, in which a therapeutic gene is introduced into the patient’s body; and an ex vivo variety, in which patients’ stem cells are harvested, equipped with a healthy version of a gene, and then infused back into the body. Orchard, like Bluebird Bio (NASDAQ: BLUE) and Avrobio, focuses on the latter approach, and has been developing gene therapies for rare immune and metabolic diseases.

In a prepared statement, Orchard said the cash will help get its three most advanced programs—which include the two GSK programs for MLD and WAS—to market.