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Ionis Pharmaceuticals

🇺🇸United States
Ownership
-
Employees
927
Market Cap
$7B
Website
Introduction

Ionis Pharmaceuticals, Inc. engages in the development and commercialization of human therapeutic drugs using antisense technology. It operates through the Ionis Core segment. The Ionis Core segment exploits a novel drug discovery platform to generate a pipeline of drugs. The company was founded by Stanley T. Crooke, David J. Ecker, Christopher K. Mirabelli, and Brett P. Monia on January 10, 1989 and is headquartered in Carlsbad, CA.

Biogen Advances Salanersen to Phase 3 Studies for Spinal Muscular Atrophy After Promising Results

Biogen's salanersen (BIIB115/ION306) demonstrated substantial slowing of neurodegeneration and clinically meaningful motor function improvements in children with spinal muscular atrophy previously treated with gene therapy.

Ulefnersen Shows Unprecedented Recovery in Young Patients with Rare FUS-ALS

Columbia University researchers report that ulefnersen, an experimental drug, demonstrated remarkable efficacy in treating FUS-ALS, a rare genetic form of ALS affecting young people.

Ionis Reports Positive Phase 3 Results for Olezarsen in Hypertriglyceridemia

Olezarsen demonstrated significant triglyceride reductions of 61% and 58% at 6 months with 80mg and 50mg monthly doses respectively in patients with moderate hypertriglyceridemia.

Ionis and Ono Forge $940 Million Deal for Sapablursen in Polycythemia Vera Treatment

Ionis Pharmaceuticals has licensed sapablursen, an RNA-targeted medicine for polycythemia vera, to Ono Pharmaceutical in a deal worth up to $940 million including $280 million upfront.

EU Approves WAINZUA (Eplontersen) for Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

The European Commission has approved WAINZUA (eplontersen), developed by Ionis Pharmaceuticals and AstraZeneca, for treating hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adult patients with stage 1 or 2 disease.

Ionis Advances Antisense Therapy for Angelman Syndrome to Phase 3 Following Promising Clinical Results

Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug.

Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments

Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.

Novartis Pushes Back Phase 3 Data Timeline for Ionis-Partnered Cardiac Therapy

Novartis announced during its Q4 2024 financial results that the timeline for Phase 3 trial data of its cardiac therapy, developed in partnership with Ionis Pharmaceuticals, will be delayed.

Novartis Extends Timeline for Pelacarsen Phase 3 Cardiovascular Trial to 2026

Novartis announces Phase 3 data for pelacarsen, an Lp(a)-lowering antisense therapy, is now expected in first half of 2026, with regulatory submissions planned for second half.

Donidalorsen Shows Sustained Efficacy in Hereditary Angioedema Prophylaxis

Donidalorsen demonstrates a sustained reduction in HAE attack rates and improved quality of life in patients, according to Phase 3 and Phase 2 clinical program analyses.

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