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Antisense battles small molecule for slice of rare lipid disorder market

Nature Biotechnology volume 31, pages 179–180 (2013)Cite this article

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“Kynamro marks the first approval of a systemically administered antisense drug in humans and has certainly validated Isis' [antisense] platform,” says Nicholas Bishop, a biotech analyst at Cowen and Company in New York. The approval also opens the door to other antisense drugs. “Certainly, the approval of Kynamro for a chronic indication reflects a comfort level with the FDA for antisense in general,” says Stanley Crooke, CEO of Isis.

Crooke and Isis have been doggedly pursuing antisense technology since 1989. In the late 1990s, many other pioneering antisense companies gave up or fell by the wayside, but Isis has built its reputation as a flagship, acquiring an almost unassailable intellectual property portfolio in antisense oligonucleotides. Isis gained the first approval of an antisense product, Vitravene (fomivirsen), in 1996 to treat retinal inflammation associated with cytomegalovirus. All has not been plain sailing since then. With poor sales of Vitravene (it is no longer available) and several clinical failures in efficacy trials, Isis has continued to hone its oligonucleotide chemistries to produce a second-generation of antisense molecules based on 2′-O-(2-methoxy) ethyl–modified ribose oligonucleotides.

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    Gunjan Sinha

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Sinha, G. Antisense battles small molecule for slice of rare lipid disorder market. Nat Biotechnol 31, 179–180 (2013). https://doi.org/10.1038/nbt0313-179

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