The first recombinant human protein made in transgenic rabbit milk was approved in October by the European Medicines Agency. Proof-of-efficacy data in human phase 2 studies were obtained for several nucleic acid therapies: OncoGenex Pharmaceuticals' custirsen and Hemispherx Biopharma's Ampligen (rintatolimod) met their endpoints, whereas Antisense Pharma's trabedersen showed only a clinical benefit. Elsewhere, results were positive for Vertex Pharmaceuticals' VX-770 in a phase 2 trial of cystic fibrosis patients.
Top ten disease grouped by pipeline size
Oncology drugs dominate the development pipeline.
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Peng, W. Drug pipeline: Q410. Nat Biotechnol 29, 101 (2011). https://doi.org/10.1038/nbt.1776
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DOI: https://doi.org/10.1038/nbt.1776
