Rare Disease Report

GSK to Transfer Gene Therapy Portfolio to Orchard Therapeutics

APRIL 13, 2018
Mathew Shanley
On Thursday, GlaxoSmithKline (GSK) announced that it will be transferring its portfolio of approved and investigational gene therapies to Orchard Therapeutics in exchange for a 19.9% stake in the company.

Under the agreed terms, GSK will also receive a seat on the company’s board, a small upfront payment, undisclosed financial considerations in the form of royalties, and commercial milestone payments related to the acquired portfolio.

The portfolio includes both approved and investigational rare disease medicines, and the agreement comes on the heels of a strategic review of the unit that was initiated last year, with which GSK decided to cut more than 30 clinical and preclinical drug development programs in an effort to improve the efficiency of its business model. According to John Lepore, Senior Vice President of the Research and Development (R&D) pipeline at GSK, the deal will allow the company to turn much of its attention to oncology, and respiratory and HIV/infectious-disease programs.

GSK will also begin work in immunoinflammation therapy areas, and “focus on building its broader cell and gene therapy platform capabilities.”

"Since we announced our intent to review these medicines, our goal has been to identify the right owner who can build on what we've already achieved," said Lepore.

The portfolio of gene therapy programs includes, most notably, (Strimvelis), which was developed alongside Fondazion Telethon and Ospedale San Raffaele. The drug was approved in Europe in 2016 as the first autologous ex vivo gene therapy for children with adenosine deaminase (ADA) severe combined immunodeficiency. The therapy consists primarily of autologous CD34+ cells transduced to express ADA.

Currently, Strimvelis is offered only at a single center in Milan, Italy, and while the drug is assumed to provide an outright cure for the condition, its commercial potential has gone unproven due to difficulty organizing cross-border reimbursement. In March 2017, the first patient was administered the drug in Milan. Its lone location for availability can be partly to blame for the delay in commercialization, per GSK’s project leader for Strimvelis, Jonathan Appleby.

As an alternative to traveling to Europe for the treatment, Orchard Therapeutics is currently developing its own treatment for ADA severe combined immunodeficiency, OTL-101, which is designed to be frozen so it can be sent to patients in any location. The company intends to submit a marketing application to the US Food and Drug Administration (FDA) later this year.

"The addition of these programs is a really big step up in terms of activity, so we are going to be looking to raise further funds through an additional private round." Orchard Therapeutics CEO Mark Rothera said in a press release. "I am very confident that we will be raising funds in the not too distant future.”

As part of the deal, the companies have agreed to exchange manufacturing, scientific and commercial knowledge. Rothera also noted that, with this revelation, Orchard could also consider going public.

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