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Ionis Pharmaceuticals

Ionis Pharmaceuticals
Formerly called
Isis Pharmaceuticals
Industry Biotechnology
Headquarters Carlsbad, United States
Products Mipomersen, Nusinersen
Website www.ionispharma.com

Ionis Pharmaceuticals (NASDAQIONS) (known as Isis Pharmeceuticals until December 2015) is a publicly traded pharmaceutical company based in Carlsbad, California, United States. The company was founded in 1989 by Stanley Crooke, a former GlaxoSmithKline head of research, with a goal to commercialize antisense therapy.

The company received its first FDA approval for an investigational new drug application in 1992, marking the first time any antisense drug was approved to be tested in humans; the IND was for Isis' genital warts drug candidate.

The company and others founded to bring antisense drugs to market faced early failures; Isis' lead compound for genital warts failed in clinical trials and Isis terminated development in 1995; by that time two other companies working on antisense, Gilead Sciences and Genta had left the field, leaving Isis, Hybridon, and Lynx Therapeutics working in the field. Gilead sold the patents it had developed around antisense to Isis.

In December 2015 Isis changed its name to Ionis, driven in part by the negative image of "Isis" generated by the terrorist group, Islamic State of Iraq and the Levant, commonly known as ISIL or ISIS.

The company's first marketed drug was fomivirsen (Vitravene) which was used in the treatment of cytomegalovirus retinitis (CMV) in immunocompromised patients, including those with AIDS. It was discovered at the NIH and was licensed and initially developed by Isis, which subsequently licensed it to Novartis. It was approved by the FDA for CMV in Aug 1998, and was the first antisense drug that was approved. Novartis withdrew the marketing authorization in the EU in 2002 and in the US in 2006. The drug was withdrawn because while there was a high unmet need for drugs to treat CMV when the drug was initially discovered and developed due to the CMV arising in people with AIDS, the development of HAART dramatically reduced the number of cases of CMV.


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