- GlaxoSmithKline plc will hand off its gene therapy portfolio to Orchard Therapeutics Ltd., divesting Strimvelis and half a dozen pipeline candidates for a near 20% stake in the U.K. biotech.
- The move completes a strategic review launched by the British pharma last summer to find a new home for its gene therapy assets. Per the deal, Orchard will also take over GSK's obligations under a 2010 collaboration with the Milan, Italy-based San Raffaele-Telethon Institute for Gene Therapy.
- GSK's decision to step away from gene therapy comes as other pharmas are deciding the time is right to invest more broadly in the field. Earlier this week, Novartis AG paid $8.7 billion to acquire AveXis Inc. and its gene replacement treatment for spinal muscular atrophy.
Chief among the assets GSK offloaded to Orchard is Strimvelis, an ex vivo stem cell gene therapy approved in Europe to treat an ultra-rare inherited immune deficiency known as ADA-SCID. Sometimes called "bubble-boy disease", ADA-SCID results in a dysfunctional immune system and usually die within the first year of life if not treated with enzyme replacement therapy.
GSK has struggled to market the pricey therapy, which costs nearly $700,000. So far, only a few patients have received treatment commercially.
In some ways, Strimvelis showcases the challenges of commercializing gene therapy. Patients are more difficult to find, while the procedure for administering an autologous treatment raises logistical hurdles. Above all, the high price tags gene therapies carry have sparked concerns about affordability and patient access.
Under new CEO Emma Walmsley, GSK is in the midst of a major overhaul of its R&D operations. Last July, the drugmaker decided to cut or otherwise divest more than 30 clinical and preclinical programs. As part of the shake-up, GSK began to search for a new owner for its gene therapy assets.
The new assets greatly broaden Orchard's pipeline and complement the company's existing development focus. Orchard is currently developing its own gene therapy for ADA SCID, for example. Two of the three clinical assets are in later-stage, registrational studies for metachromatic leukodystrophy and Wiskott Aldrich syndrome.
GSK and Orchard agreed to a transition period through the end of 2018 during which GSK will continue to conduct some clinical activities.