About Ionis_Pharmaceuticals

Ionis Pharmaceuticals is a pioneering biotechnology company specializing in **antisense therapy**, RNA interference, and CRISPR therapeutics. Founded in 1989 and based in Carlsbad, California, Ionis has been at the forefront of RNA-targeted drug development, aiming to treat serious diseases by selectively inhibiting the production of disease-causing proteins[2][3]. Originally named Isis Pharmaceuticals, the company rebranded to Ionis in 2015 to avoid association with the terrorist group ISIS. Ionis’ breakthrough came with the development of mipomersen, branded as Kynamro, a cholesterol-lowering drug approved by the FDA in 2013 after a complex partnership with Genzyme. This marked Ionis’ first major commercial success, providing significant revenue and validating its antisense technology platform[2][3]. Ionis has a robust pipeline with over two dozen drugs under development, focusing on cardiovascular, neurological, and rare diseases. Its technological advances include the use of constrained ethyl (cET) chemistry for enhanced drug potency and GalNAc conjugation, which improves liver-targeted delivery of therapies[3]. The company also founded Akcea Therapeutics, its commercial affiliate that markets drugs like TEGSEDI and WAYLIVRA in Europe and Brazil[3]. Key achievements include FDA approvals of multiple antisense therapies such as SPINRAZA (for spinal muscular atrophy), TEGSEDI, QALSODY, WAINUA, and TRYNGOLZA between 2016 and 2024, highlighting Ionis’ expanding impact in rare disease treatments[3]. In 2020, CEO Brett Monia refocused Ionis on advancing its wholly owned pipeline, emphasizing innovation and growth[3]. Ionis is recognized for its **bold scientific ambition**, lean decision-making culture, and strong commitment to corporate responsibility, including partnerships with patient advocacy groups and STEM education initiatives. This culture fosters rapid innovation and patient-centered development, positioning Ionis as

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