GlaxoSmithKline to divest rare disease gene therapies to Orchard Therapeutics

GlaxoSmithKline announced Thursday an agreement to transfer its portfolio of approved and investigational rare disease gene therapies to Orchard Therapeutics following a strategic review of the unit started last year. John Lepore, senior vice president of R&D pipeline at GlaxoSmithKline, said the deal will allow the drugmaker "to focus on building its broader cell and gene therapy platform capabilities," with a focus on oncology.

Under the agreed terms, GlaxoSmithKline will hold a 19.9-percent stake in Orchard Therapeutics, along with a seat on the company's board. GlaxoSmithKline will also receive undisclosed financial considerations in the form of royalties and commercial milestone payments related to the acquired portfolio. A spokesman for GlaxoSmithKline added that the company would also receive a small upfront payment.

The portfolio of gene therapy programmes includes Strimvelis, which 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, which consists of autologous CD34+ cells transduced to express ADA, was developed together with Fondazione Telethon and Ospedale San Raffaele.

Meanwhile, Orchard Therapeutics is developing its own treatment for ADA severe combined immunodeficiency, dubbed OTL-101, with a marketing application to the FDA targeted for later this year. Unlike Strimvelis, which can only be administered at one centre in Milan, Italy, OTL-101 is designed to be frozen so it can be sent to patients anywhere.

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The portfolio to be divested by GlaxoSmithKline also comprises two late-stage clinical programmes in ongoing registrational studies for metachromatic leukodystrophy and Wiskott Aldrich syndrome, and one clinical programme for beta thalassaemia. In addition, Orchard Therapeutics will gain rights to exclusively license three additional preclinical programmes from Fondazione Telethon and Ospedale San Raffaele upon completion of clinical proof-of-concept studies for mucopolysaccharidosis type 1, chronic granulomatous disease and globoid cell leukodystrophy.

The companies noted that under the deal, Orchard Therapeutics will assume all obligations arising from GlaxoSmithKline's 2010 agreement with the Ospedale San Raffaele and Fondazione Telethon, as well as the drugmaker's collaboration with MolMed. 

Orchard Therapeutics CEO Mark Rothera remarked "the addition of these programmes 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." Rothera said "I am very confident that we will be raising funds in the not too distant future," adding "we could also consider going public."

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