GlaxoSmithKline offloads rare disease gene therapies to Orchard Therapeutics

GlaxoSmithKline is bidding adieu to its stable of gene therapies—it is transferring the assets to Orchard Therapeutics for a 19.9% stake in Orchard and an undisclosed amount in potential milestones and royalties.

The portfolio includes the EMA-approved Strimvelis, which treats ADA-SCID, also known as “bubble boy syndrome,” a rare genetic disorder that causes immunodeficiency. Orchard will also pick up late-stage candidates for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical program for beta thalassemia. It will gain the rights to exclusively license three additional preclinical programmes from Telethon/Ospedale San Raffaele for mucopolysaccharidosis type 1 (MPS1 or Hurler syndrome), chronic granulomatous disease (CGD) and globoid cell leukodystrophy (GLD). 

The deal “strengthens Orchard’s position as a global leader in gene therapy for rare diseases” and secures “the continued development of the programs and access for patients,” GSK said in a statement. Unloading the portfolio will free Glaxo up “to focus on building its broader cell and gene therapy platform capabilities,” the company said.

“The acquisition immediately expands our primary immune deficiency and inherited metabolic disorder franchises and adds the potential for other franchises in the future,” said Orchard CEO Mark Rothera.

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“In the two late stage programs MLD and WAS for example, the clinical data are very encouraging and we look forward to continuing to progress development,” he said.

Orchard launched in 2016 with $33 million in series A money, two GSK alums among its founders and the hope to develop a drug to rival Strimvelis. Since then, the biotech reeled in another $110 million in financing, which would carry its ADA-SCID candidate through late-phase development. It also has earlier-stage programs aimed at immune deficiencies and metabolic diseases, including X-linked chronic granulomatous disease and Sanfilippo syndrome type A and type B.

Now, Reuters reports, Orchard is planning a private stock offer, with a potential IPO to follow.

GSK ran a review of its rare disease pipeline last year, announcing in July that it may sell off the assets. While the British pharma has “been heavily invested in this area, we believe there is someone else who can best ensure the commercial availability of these medicines for patients,” the company said at the time.

If this feels like déjà vu, that’s because Pfizer did the same thing with its CAR-T pipeline last week. It signed over the rights to 17 assets—only one of which has been tested in humans—to Allogene Therapeutics, a new biotech helmed by former Kite executives Arie Belldegrun and David Chang. The Big Pharma had previously licensed the programs from Cellectis in 2014 and 2015 in a deal that could have been worth up to $2.9 billion.