Ionis Pharmaceuticals

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Ionis Pharmaceuticals is a commercial-stage biotech leader with a mission to discover and deliver life-changing RNA-targeted medicines. The company has achieved multiple industry firsts, including the first approved antisense drug, and has successfully transitioned from a research-focused entity to a fully integrated organization with its own commercial capabilities. Its strategy centers on independently launching medicines in neurology and cardiology while selectively partnering in other areas, backed by a deep, late-stage pipeline and a sustainable technology platform.

Proprietary antisense oligonucleotide (ASO) platform enabling precise RNA-targeting for protein modulation, built on iterative chemistry innovations including phosphorothioate backbones, 2'MOE modifications, and ligand conjugation (LICA) for tissue-specific delivery.