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The past 5 years have seen an unprecedented rate of discovery of genes that cause rare diseases and with it a commensurate increase in the number of diagnosable but nevertheless untreatable disorders. Here, we discuss the increasing opportunity for diagnosis and therapy of rare diseases and how to tackle the associated challenges.
A pioneering Merck & Co.-funded study is set to explore the ability of the microbiome to boost immuno-oncology therapy outcomes, and other companies are quickly gearing up to enter this clinical space.
With Alnylam's rare disease drug candidate patisiran nearing the regulatory finish line, the RNA interference community is now turning its attention to next-generation delivery technology to solidify the future of the emerging modality.
Fiona Marshall — co-founder of Heptares, former chief scientific officer of Sosei and soon to be lead of Merck & Co.'s new research facility in London — looks back on 30 years of GPCRs, and looks forward to the emerging therapeutic opportunities these targets still offer.
In 2011, AstraZeneca set out to improve its research and development (R&D) productivity by focusing decision-making using a framework with five technical determinants: the right target, right tissue, right safety, right patient and right commercial potential. Here, Pangalos and colleagues describe the progress made using this '5R framework', with metrics indicating improved success rates, and discuss where the approach has failed and the lessons learned.
The contribution of genomics to drug discovery and development so far has not yet lived up to the initial high expectations. Goldstein and colleagues discuss the reasons for the limited progress and review how recent advances — particularly in oncology and rare genetic diseases — may enable precision medicine strategies to harness the therapeutic potential of genomic knowledge.
Antibody therapeutics are now established as a major drug class. Here, Carter and Lazar comprehensively discuss current and emerging platforms and technologies for antibody therapeutics, with an emphasis on approaches that could extend their therapeutic applications, including antibody–drug conjugates, bispecific antibodies and antibody engineering to enable more effective delivery.
