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Jan 7, 2013

Shire Rare-Disease Partnerships Expand

  • Shire today broadened its research partnership efforts aimed at rare disease, launching a new alliance with ethris aimed at developing RNA-based therapeutics for rare diseases, and expanding a year-long monoclonal antibody therapeutics collaboration with arGEN-X to include a second undisclosed rare disease.

    Under the alliance with German-owned ethris, Shire will develop mABs using its partner’s RNA modification technology platform developed by ethris and its co-founders, Carsten Rudolph, Ph.D., and Christian Plank, Ph.D. The platform produces Stable and Non-Immunogenic Messenger RNA (SNIM®-RNA) molecules for use in protein replacement therapies to treat monogenic genetic diseases.

    “We are aiming to develop an RNA modification and delivery technology that efficiently results in protein replacement in relevant cellular populations, providing hope to patients suffering from genetic diseases which have devastating clinical outcomes,” Dr. Plank, ethris’ CSO, said in a statement.

    Ethris did not disclose financial details of its partnership with Shire, whose Human Genetic Therapies (HGT) unit develops and markets drugs for various rare diseases.

    SNIM-RNA showed promise in a preclinical study in a mouse model of the fatal human hereditary disease surfactant protein B deficiency, published in January 2011 in Nature Biotechnology: “ethris envisages a strong potential of the SNIM-RNA platform in regenerative medicine and in the treatment of metabolic disorders,” the company said in a statement at the time.

    In the partnership with arGEN-X of the Netherlands, Shire is using its partner’s Superior Immunodiversity with Minimal Protein Lead Engineering (SIMPLE) Antibody™ discovery platform to create human antibody therapeutics designed to address diverse rare and unmet diseases. Shire has the option to license the most promising leads for further preclinical development and commercialization worldwide.

    Shire has agreed will pay fees, milestones, and royalties on product sales to arGEN-X, which is receiving undisclosed amounts of R&D funding and preclinical success payments, in return for awarding exclusive options to develop and commercialize products.

    arGEN-X made progress with another commercial partner last October, when it granted RuiYi (formerly Anaphore) a worldwide exclusive license to develop and commercialize ARGX-109, an anti-IL-6 SIMPLE Antibody™ discovered and developed by arGEN-X.

    According to arGEN-X, ARGX-109 has outstanding neutralization potency for the cytokine IL-6, widely implicated in cancer and autoimmunity. ARGX-109 is the first of five preclinical stage human antibody candidates developed by arGEN-X from its SIMPLE Antibody™ platform in the three years since its start of operations

    The following month, arGEN-X filed a first CTA for ARGX-110, its most advanced SIMPLE Antibody program modulating CD70 via a unique mode of action in hematological and virally-induced solid tumors, as well as in autoimmunity. A second CTA for ARGX-111, a novel anti-c-Met antibody to treat diverse solid tumors, is on track for filing this year.  


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