Published On: Sat, Apr 14th, 2018

GSK offloads rare disease gene therapy drugs to Orchard Therapeutics

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Pharma acquisition news : British drug company GlaxoSmithKline (GSK) has agreed to transfer its rare disease gene therapy drugs to Orchard Therapeutics for an equity stake of 19.9% and a seat on the board of the UK gene therapy company.

For some time now, GSK has been mulling to sell the rare disease drug portfolio as part of a strategic review initiated in July 2017. The British drug company is inclined towards enhancing its pharma pipeline with focus on developing drugs for respiratory, HIV/infectious diseases, oncology and immuno-inflammation.

Its rare disease gene therapy drugs that will be transferred to Orchard Therapeutics include Strimvelis – an EMA approved bubble baby syndrome gene therapy along with certain investigational gene therapies.

As per the deal with Orchard Therapeutics, GSK will earn from royalties and commercial milestone payments related to the rare disease gene therapy drugs it has transferred.

GSK’s latest deal comes within days of its $13bn proposal to buy out the stake of Novartis in their consumer healthcare joint venture.

GSK's corporate headquarters in Brentford, London

GSK’s corporate headquarters in Brentford, London. Photo courtesy of GlaxoSmithKline plc.

John Lepore – Senior Vice President of GSK R&D pipeline said: “GSK is proud of the advances we have achieved in collaboration with the cell and gene therapy pioneers at Ospedale San Raffaele, Fondazione Telethon and MolMed in Milan.

“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, and can advance these important medicines for patients, allowing GSK to focus on building its broader cell and gene therapy platform capabilities.

“Orchard are committed to patient access, and we’re confident that this agreement combined with the ongoing relationship between the two companies will support the progression of these valuable programmes to enable them to benefit patients.”

For Orchard Therapeutics, the GSK deal consolidates its global presence in gene therapies for rare diseases.

The deal includes GSK’s gene therapy drugs for metachromatic leukodystrophy and Wiskott Aldrich syndrome – currently in late-stage development and an investigational therapy for beta thalassaemia.

The only approved drug to be acquired by Orchard Therapeutics is Strimvelis, which is an autologous ex vivo gene therapy drug, indicated for the treatment of bubble baby syndrome, which is technically known as adenosine deaminase severe combined immunodeficiency (ADA-SCID) in children.

Orchard Therapeutics will also bag exclusive rights to license an additional three preclinical programs from Telethon/Ospedale San Raffaele, post completion of clinical proof of concept trials for Hurler syndrome, chronic granulomatous disease and globoid cell leukodystrophy.

Mark Rothera – CEO of Orchard Therapeutics said: “Acquiring this portfolio further advances Orchard’s vision to be a global, fully integrated company leading the field of gene therapy for rare diseases.

“The acquisition immediately expands our primary immune deficiency and inherited metabolic disorder franchises and adds the potential for other franchises in the future. At Orchard, we are committed to transforming the lives of patients with rare diseases through innovative gene therapies.”

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