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The development of therapies that are capable of safely achieving sizeable and sustained body weight loss has proved tremendously challenging. Here, Müller et al. provide an overview of the history of anti-obesity drug development, focusing on lessons learned, ongoing challenges and recent advances in the field.
The NIH and FDA’s newly launched 27-member public–private partnership will spend US$76 million addressing the hurdles of AAV-based gene therapies for ultra-rare diseases.
Susan Galbraith, head of Oncology Research & Development at AstraZeneca, discusses the opportunities oncologists can’t walk away from, including HER2, antibody–drug conjugates and earlier intervention.
Biogen and Ionis’s SOD1-antisense oligonucleotide tofersen failed a first phase III trial, raising questions about the next steps for this drug and for future ALS trials.
Lack of predictive preclinical models is one of the reasons for the high rate of attrition in oncology drug development. This Review discusses the issues in preclinical-to-clinical translatability of molecularly targeted cancer therapies and the need to better align tumour biology in patients with preclinical model systems.
The unfolded protein response (UPR) aims to relieve endoplasmic reticulum (ER) stress and restore protein homeostasis, but also contributes to disease. Here, Marciniak et al. assess small molecules that target ER stress and the UPR, highlighting those diseases in which the role of the UPR and its therapeutic modulation have been most well studied.
