Nature Reviews Drug Discovery

Strategies for delivering value from digital technology transformation ()
Many organizations are attempting to harness emerging digital technologies and the surge in the amount of health-related data to drive advances in the development and use of medicines. Focusing on just a few well-proven and readily available strategies could enable such organizations to quickly realize greater value from data and digital technologies.
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2016 FDA drug approvals ()
FDA approval count fell last year, despite a steady regulatory filing rate.
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2016 EMA drug approval recommendations ()
The European Medicines Agency (EMA) recommended approval for 27 new therapeutic agents in 2016, down from 39 in 2015 and 40 in 2014.The EMA's count includes small molecules, antibodies, biologics, blood products, cellular therapies and vaccines, and so cannot be compared directly with
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FDA approves splice-modulating drug ()
The FDA approved Biogen and Ionis Pharmaceuticals' nusinersen for spinal muscular atrophy (SMA) in December 2016.SMA is a hereditary muscle-wasting disease caused by loss-of-function mutations in the survival motor neuron 1 (SMN1) gene. Nusinersen is an antisense oligonucleotide that modulates the splicing
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Cancer reproducibility project yields first results ()
In 2011, Bayer researchers made a splash with news that they could only replicate 25% of the preclinical academic projects that they took on (Nat. Rev. Drug Discov.10, 712; 2011). Amgen fared even worse when trying to recreate the findings from
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Ebola vaccine success ()
Four years since the onset of the 2013–2016 Ebola outbreak, researchers have solid clinical evidence that a vaccine can reduce the spread of the deadly haemorrhagic fever.Researchers set up an open-label phase III trial designed to test the vaccine in people who may have
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Market watch: Value of 2016 FDA drug approvals: reversion to the mean? ()
2014 and 2015 were extraordinary years for the biopharmaceutical industry, with records set in both the number and value of the new therapeutic drugs (NTDs) approved by the FDA. With 2016 figures now on the books (see Nat. Rev. Drug Discov., 16, 73–76;
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Regulatory watch: Outcomes of early health technology assessment dialogues in medicinal product development ()
The development of medicinal products has been shifting towards a value-driven model. However, although companies need to engage with health technology assessment (HTA) bodies to inform effective strategies to gain market access for their new medicinal products, HTA scientific advice (SA) procedures are not yet
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John Jenkins ()
When John Jenkins started working at the FDA in 1992, the agency had just created its accelerated approval pathway, had only approved a single monoclonal antibody and did not have a stand-alone oncology division. Over the subsequent 25 years, he has helped transform the agency, shaping Prescription Drug User Fee Acts, building more flexibility into the regulatory system, getting grilled by Congress about controversial approvals and navigating the way through difficult safety decisions. Last month, he resigned from his role as Director of the FDA's Office of New Drugs to take on new challenges. He spoke with Asher Mullard about approvals standards, breakthrough therapy designation and regulatory science hurdles.
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The immuno-oncology race: myths and emerging realities ()
This article analyses the huge volume of clinical trial activity for immune checkpoint inhibitors, and discusses the development of the market and strategic trends for immuno-oncology therapies in general.
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Genetic disorders: Steps towards epigenetic therapy for PWS ()
Prader–Willi Syndrome (PWS) is an imprinting disorder caused by a deficiency of paternally expressed genes along a specific region of chromosome 15 (15q11–q13) and is characterized by neonatal hypotonia, failure to thrive, childhood onset obesity and intellectual disability. Current treatment of PWS largely consists of
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Autoimmune diseases: Inhibitor of adaptor protein shows self-antigen selectivity ()
Patients suffering from autoimmune diseases are usually treated with immunosuppressive drugs, leaving them prone to infection. Now, reporting in Science Translational Medicine, Alarcon and colleagues show that a small molecule inhibitor of the adaptor protein NCK, which acts downstream of the T cell receptor
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Receptor pharmacology: Picking the pocketome for orphan receptor ligands ()
G protein-coupled receptors (GPCRs) are a well-established class of drug target, but several members of this family, so-called orphan GPCRs, remain pharmacologically intractable owing to lack of knowledge about their cognate ligands or their structures. Now, Ngo et al. report a new approach to
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Cancer: Tumour vessel normalization takes centre stage ()
Tumour vasculature is known to be abnormal: whether this feature should be exploited or corrected therapeutically has been the subject of debate. Two reports in Cancer Cell suggest that tumour vessel normalization — either by altering angiogenesis through the TIE2 (also known as angiopoietin
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Alzheimer disease: Identification of novel Aβ inhibitors ()
Targeting amyloid-β (Aβ) aggregation and accumulation has been pursued as a major potential therapeutic strategy against AD, but no compound has yet gained regulatory approval. Habchi et al. now describe the use of a quasi-structure-based and kinetics-based drug discovery approach to identify a pool
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Neurodegenerative disease: Pituitary adenylate cyclase activator ameliorates SBMA ()
Post-translational modifications, such as phosphorylation, modify the toxicity of the polyglutamine (polyQ) expansion in the androgen receptor (AR) (polyQ-AR), which occurs in spinobulbar muscular atrophy (SBMA). Here, Polanco et al. show in cell models that cyclin-dependent kinase 2 phosphorylates polyQ-AR specifically at
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HIV: CRISPR screen identifies novel therapeutic targets ()
Host proteins, termed host dependency factors (HDFs), are crucial for productive HIV infection but dispensable for cellular viability, thereby representing promising therapeutic targets. Here, Park et al. conduct a CRISPR-based genetic screen in a CD4+ T cell line to identify five HDFs
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Anticancer drugs: The fat controller ()
The ability to target cells with metastatic potential is of crucial importance in cancer treatment, which is why new results from the team led by Aznar Benitah, who has identified a subpopulation of cells with high metastatic potential, are exciting news.The authors were interested
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Epilepsy: HSP90 inhibition suppresses seizures ()
Loss of the glutamate transporter GLT1 (also known as SLC1A2) occurs in temporal lobe epilepsy (TLE), but the mechanisms mediating GLT1 degradation are not understood. Here, Sha et al. report upregulated expression of HSP90β in reactive astrocytes of human epileptogenic tissue and in mouse
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Cornerstones of CRISPR–Cas in drug discovery and therapy ()
The recent development of CRISPR–Cas systems as easily accessible and programmable tools for genome editing and regulation is spurring a revolution in biology. Paired with the rapid expansion of reference and personalized genomic sequence information, technologies based on CRISPR–Cas are enabling nearly unlimited genetic manipulation,
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Induced protein degradation: an emerging drug discovery paradigm ()
Small-molecule drug discovery has traditionally focused on occupancy of a binding site that directly affects protein function, and this approach typically precludes targeting proteins that lack such amenable sites. Furthermore, high systemic drug exposures may be needed to maintain sufficient target inhibition in vivo,
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Induced pluripotent stem cell technology: a decade of progress ()
Since the advent of induced pluripotent stem cell (iPSC) technology a decade ago, enormous progress has been made in stem cell biology and regenerative medicine. Human iPSCs have been widely used for disease modelling, drug discovery and cell therapy development. Novel pathological mechanisms have been
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DNA-encoded chemistry: enabling the deeper sampling of chemical space ()
DNA-encoded chemical library technologies are increasingly being adopted in drug discovery for hit and lead generation. DNA-encoded chemistry enables the exploration of chemical spaces four to five orders of magnitude more deeply than is achievable by traditional high-throughput screening methods. Operation of this technology requires
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