GlaxoSmithKline (GSK) said today it will hand off its rare disease gene therapy portfolio—including its European-approved gene therapy Strimvelis™—to two-year-old startup Orchard Therapeutics in return for taking a 19.9% stake in the acquirer, as well as undisclosed milestone payments and royalties, and a seat on Orchard’s board.

The portfolio includes two late-stage clinical programs in ongoing registrational studies—one indicated for metachromatic leukodystrophy (MLD) and the other, Wiskott Aldrich syndrome (WAS).

Orchard will also acquire rights to exclusively license three additional preclinical programs from Ospedale San Raffaele (OSR) and Fondazione Telethon (Telethon), which developed Strimvelis, upon completion of clinical proof-of-concept studies for mucopolysaccharidosis type 1 (MPS1, or Hurler syndrome), chronic granulomatous disease (CGD), and globoid cell leukodystrophy (GLD).

Also in the portfolio are a clinical program for beta thalassaemia—as well as Strimvelis (autologous CD34+ cells transduced to express adenosine deaminase, or ADA), which received European Commission approval in 2016 as the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Strimvelis was developed by OSR and Telethon through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET). The therapy was advanced by GSK through a strategic collaboration formed in 2010 between the company, OSR, and Telethon. Orchard’s co-founders include CMO Andrea Spezzi, M.D., FFPM, who previously served as VP and medicine development leader at GSK’s Rare Diseases Unit.

“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,” John Lepore, M.D., GSK’s SVP, R&D pipeline, said in a statement.

MolMed is a gene therapy developer that had been partnering with GSK, and separately announced today it will assume the pharma’s role in the collaboration with MolMed, as well as in the partnership with OSR and Telethon.

However, GSK will continue to conduct unspecified activities through the end of 2018 “in order to support a smooth transition of these programs with minimal disruption to the projects,” Orchard added.

Under CEO Emma Walmsley, who succeeded Sir Andrew Witty as of March 31, 2017, GSK has overhauled its R&D pipeline, last year terminating more than 30 clinical and preclinical programs deemed by the company as “unlikely to generate sufficient returns.”

GSK also named a new CSO and president, R&D, Hal Barron, M.D., who previously served as president of R&D at Calico (California Life Sciences LLC), a Google-backed company launched in 2013 to focus on the genetics and biology of aging and the development of approaches that can slow aging and counteract age-related diseases. Earlier he advanced from a clinical scientist to holding executive positions at Genentech and later its parent company, Roche, where he was EVP, head of Global Product Development, and CMO.

“The Right Owner”

“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,” Lepore added. “Orchard is 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 programs to enable them to benefit patients.”

GSK and Orchard have agreed to exchange manufacturing, technical, and commercial insights and learning on the development of gene therapies, with the goal of ensuring the success of the portfolio.

GSK said it remains committed to developing platform capabilities in cell and gene therapies, but will narrow its focus to oncology, one of the pharma giant’s 12 key therapeutic areas of interest.

In addition to oncology, GSK’s key areas of therapeutic interest include bioelectronics R&D; biopharmaceuticals technologies and processes; consumer healthcare; immuno-inflammation; infectious diseases, including bacterial, viral, and parasitic infections; metabolic and cardiovascular; neglected tropical diseases; neurosciences; ophthalmology; respiratory; and vaccines. The company adds that it will consider partnering in other therapeutic areas with developers of medicines, technology, or assets of interest.

Launched in 2016, Orchard focuses on developing gene therapies for primary immune deficiencies and inherited metabolic disorders. Orchard’s pipeline is anchored by OTL-101, an autologous ex vivo lentiviral gene therapy in development to treat ADA-SCID, and includes clinical and preclinical gene therapies for immune deficiencies that include X-linked chronic granulomatous disease (X-CGD) and metabolic diseases, including MPS-IIIA and MPS-IIIB (Sanfilippo syndrome type A and type B).

In December 2017, Orchard completed an oversubscribed $110 million Series B financing, saying that proceeds would be used to advance the development of OTL-101, as well as expand the company’s manufacturing infrastructure and business development activity. Baillie Gifford and ORI Capital co-led the financing, with new investments from Temasek and Cowen Healthcare Investments, Juda Capital, Pavilion Capital, RTW Investments, Agent Capital, 4BIO Capital, as well as previous investors F-Prime Capital and UCL Technology Fund.

The financing brought to $160.5 million the amount of funding raised by Orchard, according to Crunchbase.


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