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February 15, 2018

Oxford BioMedica, Bioverativ to Develop Lentiviral Vectors for Hemophilia Gene Therapy

  • Oxford BioMedica said today it will partner with Bioverativ to develop and manufacture lentiviral vectors designed to treat hemophilia, through a collaboration that could generate up to $105 million-plus for Oxford BioMedica.

    Bioverativ—which is being acquired by Sanofi for approximately $11.6 billion—has agreed to license Oxford BioMedica’s LentiVector® Enabled technology, as well as its industrial-scale manufacturing technology.

    LentiVector is a lentiviral-based gene delivery system designed to overcome safety and delivery challenges associated with earlier-generation vectors. According to Oxford BioMedica, the platform’s advantages include large therapeutic payloads (up to 9 kb), permanent modification of dividing and nondividing cells, and no pre-existing immunity

    The platform can also integrate genes into nondividing cells, including in the brain and retina, with the company citing long-term studies suggesting gene expression may be maintained indefinitely—offering the prospect of permanent therapeutic benefit following a single administration.

    LentiVector is also a research tool, with applications in transgenesis, stem cell manipulation, somatic disease models, target validation, and gene discovery.

    Oxford BioMedica CTO James Miskin, Ph.D., told GEN last year that Oxford BioMedica developed its vector manufacturing capacity to its current scale through £7.7 million ($10.8 million) in catalytic funding, which was provided partly as a grant and partly as a loan by the U.K. government’s Advanced Manufacturing Supply Chain Initiative (AMSCI).

    Bioverativ has agreed to pay Oxford BioMedica $5 million upfront, up to $100 million in payments tied to achieving unspecified milestones, and undisclosed additional royalties on net sales of Bioverativ lentiviral hemophilia products.

    Bioverativ also agreed to fund process development and scale-up activities for its lentiviral vector hemophilia products at Oxford BioMedica.

  • Preclinical Gene Therapies

    According to its website, Bioverativ’s pipeline includes preclinical gene therapies for hemophilia A and hemophilia B that are being jointly developed through a collaboration with San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET). The partners are applying SR-TIGET’s advanced lentiviral gene transfer technology, continuing a collaboration launched in 2015 by Biogen, from which Bioverativ was spun out last year

    "Today's news demonstrates the value of our LentiVector Enabled platform and our leading capabilities and reputation in the industrial scale-up for lentiviral vectors for clinical and commercial supply,” Oxford BioMedica CEO John Dawson said in a statement. “Bioverativ's investment in hemophilia gene therapy underlines the potential of lentiviral vectors for use for in vivo gene therapy.

    Dawson added that the agreement with Bioverativ “demonstrates Oxford BioMedica's strategy of building multiple partnerships with leaders in their therapeutic categories and will support the Group's continued growth.”

    The Bioverativ deal comes seven months after Oxford BioMedica inked a supply agreement of comparable value with Novartis.

    Oxford BioMedica agreed in July 2017 to deliver commercial and clinical supplies of lentiviral vectors for Novartis’ chimeric antigen receptor T-cell (CAR-T) treatment KymriahTM (tisagenlecleucel) and other undisclosed CAR-T products. In return, Novartis agreed to pay Oxford BioMedica up to $100 million over three years—of which $10 million was to be paid upfront. The agreement can be extended to five years if both companies approve.

    Kymriah became the first CAR-T treatment to win FDA approval in August.

    In addition to Novartis and Bioverativ, Oxford BioMedica has signed partnership agreements with Sanofi, GC LabCell, GlaxoSmithKline, and Immune Design.

    Amy Walker, an analyst with Peel Hunt, told Reuters the deal with Bioverativ cemented her view that Oxford BioMedica’s technology was “at the forefront of the gene and cell therapy revolution.”

    Headquartered in Waltham, MA, Bioverativ focuses on developing therapies for hemophilia and other rare blood disorders and was created last year following Biogen’s spinout of its global hemophilia business.

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