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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.
Feng Zhang discusses the expanding gene-editing toolkit, the need for better delivery systems and the reprogrammable biological technologies that remain to be discovered.