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Volume 16 Issue 11, November 2017

Comment

  • Scientific advances, in combination with government incentives and commercial opportunity, have fuelled strong investment in orphan drugs, resulting in many innovative therapies. Here, we discuss the approach of the FDA to a range of issues that remain crucial to maintaining this momentum, such as the use of the totality of evidence in evaluating orphan drugs.

    • Nina L. Hunter
    • Gayatri R. Rao
    • Rachel E. Sherman
    Comment

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News and Analysis

  • The FDA is weighing the fate of Spark Therapeutics' voretigene neparvovec, potentially the first among a wave of new gene therapies.

    • Chris Morrison
    News and Analysis
  • CARB-X, a public–private partnership aimed at bolstering the antibiotic pipeline, funded a diverse set of 17 early-stage drug development projects in its first year.

    • Asher Mullard
    News and Analysis
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News in Brief

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Biobusiness Briefs

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An Audience With

  • Nora Volkow, director of the National Institute on Drug Abuse (NIDA), discusses her ambitious aim of halving the time needed to develop non-opioid analgesics and anti-addiction drugs.

    An Audience With
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From the Analyst's Couch

  • This analysis of US drug launches from the past decade indicates that commercial success has become more difficult to achieve, owing to factors such as increased competition, greater challenges in achieving therapeutic differentiation and payer pressures.

    • Nicholas Donoghoe
    • Jon Duane
    • George Xu
    From the Analyst's Couch
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Research Highlight

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In Brief

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Review Article

  • New therapies for idiopathic pulmonary fibrosis (IPF) are urgently needed. Here, Moraet al. discuss our current understanding of the mechanisms driving IPF, highlighting the parallels between fibrosis and ageing. Potential avenues for drug discovery and development are described, including progress made and current obstacles.

    • Ana L. Mora
    • Mauricio Rojas
    • Moises Selman
    Review Article
  • WD40 repeat (WDR) domain-containing proteins are involved in numerous protein complexes that have been linked to disease. Schapira and colleagues describe recent advances in targeting WDR domains with small molecules to potently inhibit protein–protein interactions, and discuss the potential for the diversity and druggability of WDR domains to open up new pathways for therapeutic intervention.

    • Matthieu Schapira
    • Mike Tyers
    • Cheryl H. Arrowsmith
    Review Article
  • G protein-coupled receptors (GPCRs) are key drug targets. In this article, Marshall and colleagues discuss the progress made towards generating GPCR-targeting antibodies, including which antigen formats and antibody platforms have been most successful. They review the current pipeline and outline outstanding challenges in antibody generation.

    • Catherine J. Hutchings
    • Markus Koglin
    • Fiona H. Marshall
    Review Article
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Corrigendum

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Erratum

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Corrigendum

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