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- Ionis Pharmaceuticals, Inc.
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Overview
Company Summary
Ionis Pharmaceuticals, Inc. is a biopharmaceutical company specializing in the development and commercialization of RNA-targeted therapeutics. The company utilizes its proprietary antisense technology platform to design and develop drugs that can modulate the production of disease-causing proteins.
Ionis Pharmaceuticals focuses on addressing a wide range of diseases, including rare genetic disorders, cardiovascular diseases, neurological disorders, and cancer. The company's approach involves targeting specific disease-causing genes and using antisense oligonucleotides (ASOs) to inhibit the translation of these genes into proteins.
The ASOs developed by Ionis Pharmaceuticals are unique because they can be tailored to target specific genes and work at the cellular level to prevent the production of harmful proteins. By modulation of protein production, Ionis aims to slow or stop the progression of diseases, alleviate symptoms, and potentially provide substantial medical benefits to patients.
The company has a robust pipeline of drug candidates at various stages of development. Some of their notable products include Spinraza (nusinersen), which received FDA approval for the treatment of spinal muscular atrophy (SMA), and Tegsedi (inotersen), approved for the treatment of hereditary transthyretin amyloidosis (hATTR).
Ionis Pharmaceuticals also collaborates with several pharmaceutical companies, leveraging their technology platform to develop drugs in partnership. These collaborations enable the company to expand its reach and develop novel therapeutics for various diseases.
In summary, Ionis Pharmaceuticals is a biopharmaceutical company that utilizes antisense technology to develop RNA-targeted therapeutics for the treatment of genetic disorders, cardiovascular diseases, neurological disorders, and cancer. Their unique approach of modulating protein production has the potential to offer new treatment options for patients suffering from various illnesses.