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Sean Harper, Amgen's Chief Scientific Officer, talks about how human genetic data can be used to avoid costly failures, prosecute programmes more effectively and discoverde novodrug targets.
STING, RIG-I and NLRP3 agonists might increase the effectiveness of immuno-oncology checkpoint inhibitors, while antagonists of these targets offer an anti-inflammatory bonus.
Numerous challenges arise when developing targeted therapies for diseases comprising low-frequency molecular subtypes. In this article, we describe a pragmatic, science-based regulatory policy for the development and approval of targeted therapies in such cases.
The development of anticancer therapies is evolving with the advent of novel targeted drugs. In this Comment, I summarize some of the key changes and discuss the various choices for the optimal development of a new generation of cancer therapeutics.
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.
