Through scientific research and educational resources, we are committed to pioneering new markets and changing the standard of care. With a novel RNA-targeted drug discovery platform called “antisense,” we are tackling a range of diseases across a number of therapeutic areas.
What is a Clinical Study?
A clinical trial is a medical research study designed to assess whether or not a new drug or treatment is well-tolerated and effective in treating people with a particular disease.
A clinical trial is a medical research study designed to assess whether or not a new drug or treatment is well-tolerated and effective in treating people with a particular disease.
Strict regulations govern the development and approval of new drugs.
All new drugs that reach the human stage are required to go through an orderly series of clinical trial steps called phases. All clinical trial phases involve thorough follow-up and assessment of the effects of the new drug in human volunteer participants. Researchers design each phase to address a different question.
Early trial phases often involve assessment of the effects and the safety of the drug in healthy volunteers. In later phases, the effects of the drug are evaluated in patients with the disease for which the drug is intended to treat. In some trials, the new drug is compared to a sham drug, called a placebo. In others, the new drug may be compared to existing treatments, to check whether the new drug offers advantages. In all clinical trial phases researchers monitor all participants carefully and regularly to gather data about the effects of the drug.
Some patients with serious diseases may be keen to try new drugs that offer them hope of improvement or cure and may find the intricacies of this regulated process frustrating. But researchers and pharmaceutical companies are legally obliged to comply with these regulations in order to ensure that there is optimal patient safety at every stage and that new drug efficacy and safety is evaluated by rigorous scientific standards.
Patient Experiences
At Ionis, our efforts begin and end with the patient in mind.
At Ionis, our efforts begin and end with the patient in mind.
From discovery to development to getting medicines into the hands of those who need it, we are inspired by the needs of the patient communities we serve and fueled by the promise of our science. Every aspect of what we do is grounded in patient perspectives.
Take a closer look at some of the people who inspire our work by clicking here.
Contact Ionis
Corporate Information
Ionis Pharmaceuticals, Inc.
2855 Gazelle Court Carlsbad, CA 92010