Volume 20 Issue 12, December 2021

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.

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.

Although checkpoint inhibitor blockade has been extremely successful in certain types of cancer, harnessing the immune system for immunosurveillance has met with numerous failures. In this Review, Mak and colleagues discuss the lessons learned from these failures, and how to incorporate the growing understanding of immuno-oncology into potential therapies, including combinations with existing checkpoint inhibitors.

Diabetes is a substantial and increasing health concern. In this Review, Lickert and colleagues discuss the progress made in developing insulin-producing islets using in vitro methods, including which aspects need to be improved in order to use these islets as transplants. Using these islets in laboratory settings could further our understanding of pancreatic function and the mechanisms underlying diabetes.

The design of cell-based therapeutics with synthetic biology is a rapidly growing strategy in medicine for the development of effective treatments for a variety of diseases. This article discusses advances in synthetic biology approaches to programme living cells with therapeutic functions as well as challenges for their development.