Ionis Pharmaceuticals leading company in RNA-targeted drug discovery and development with a pipeline of first-in-class or best-in-class medicines with the potential to provide high value for patients with significant unmet medical needs. We have created an efficient and broadly applicable drug discovery platform. Using this platform, we believe that we are revolutionizing medicine with the goal to improve the quality of, and save, lives. They have discovered and are developing three potentially transformational drugs, nusinersen, IONIS-TTRRx and volanesorsen, which we believe are close to commercialization. We designed each of these three drugs to treat patients with orphan diseases who have limited or no therapeutic options. In total, we are developing these three drugs for six different patient populations. In 2015, we completed target enrollment in the first pivotal Phase 3 study for each of these three drugs, and we anticipate reporting data from these studies in the first half of 2017. We designed nusinersen to treat patients with spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. We designed IONIS-TTR Rx to treat patients with transthyretin amyloidosis, or TTR amyloidosis, a fatal disease in which patients experience progressive buildup of amyloid plaque deposits in tissues throughout the body, including peripheral nerves, heart, intestinal tract, kidney and bladder. We designed volanesorsen to treat patients with diseases associated with extremely high levels of triglycerides, including patients with two severe and rare genetic conditions called familial chylomicronemia syndrome, or FCS, and patients with familial partial lipodystrophy, or FPL. We anticipate that the data from their pivotal Phase 3 studies of these drugs, if positive, will support global regulatory filings for each drug. We believe that the significant unmet medical need and the severity of these diseases could warrant a rapid path to market. Already their partners for these programs, Biogen for nusinersen and GSK for IONIS-TTR Rx , are preparing to commercialize these drugs. Our wholly owned subsidiary, Akcea Therapeutics Inc., or Akcea, is preparing to commercialize volanesorsen. All three companies are engaging in pre-commercialization activities to understand the patient journey, build disease awareness with physicians and patients and develop their launch plans. We believe all three of these drugs have the potential to reach the market in the next several years.

Research Grants 54 show all






Patents 11,421show all

  • 3,393
    C12N - Microorganisms or enzymes
  • 2,982
    A61K - Preparations for medical, dental, or toilet purposes
  • 1,611
    C07H - Sugars
  • 1,351
    Y02P - Climate change mitigation technologies in the production or processing of goods
  • 1,124
    C12Q - Measuring or testing processes involving enzymes, nucleic acids or microorganisms
  • 932
    C07K - Peptides
  • 695
    C12Y - Enzymes
  • 625
    G01N - Investigating or analysing materials by determining their chemical or physical properties
  • 309
    C07D - Heterocyclic compounds
  • 305
    C07F - Acyclic, carbocyclic or heterocyclic compounds containing elements other than carbon, hydrogen, halogen, oxygen, nitrogen, sulfur, selenium or tellurium

Clinical Trials 185show all

76Phase 246Phase 323Phase 118N/A10Phase 1/Phase 28Other

SEC Filings show all





Contact Information

2855 Gazelle Court
Carlsbad, CA 92010
United States


Total FundingEmployeesLast Funding DateStatus

Crunchbase Investment Rounds

DateAmountRoundValuationLead InvestorOther Investors
2012-08-27$201,250,000Post Ipo Debt

SEC Form D Funding Events

2006-04-25Unknown Unknown Other (Paper Filing)

Key Executives

  • Stanley T Crooke
    Executive Officer, Director
  • Spencer R Berthelsen
  • Joseph Klein Iii
  • Frederick T Muto
  • B Lynne Parshall
    Executive Officer, Director
  • John C Reed
  • Joseph H Wender
  • C Frank Bennett
    Executive Officer
  • Richard S Geary
    Executive Officer
  • Brett P Monia
    Executive Officer