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Ionis Innovation

Optimizing antisense

Because the building blocks of antisense therapies are constant, we are able to realize efficiencies from every treatment we move forward and apply those learnings to improve our next potentially life-altering treatment.

 

Ionis’ antisense technology is:

 

  • SpecificWe design our antisense therapies to selectively target one and only one gene product. The more selectivity a drug has for its target the better the drug.
  • AdaptableThe building blocks of antisense technology are constant across all antisense therapies. Only the “address” to which the therapy is targeted changes with each new medicine we make.
  • BroadOur antisense therapies can be precisely created for any target, including targets that have long been considered “un-druggable” by traditional small molecule and antibody therapies.
  • Active & FlexibleAntisense therapies are single-stranded molecules, allowing them to bind directly to mRNA.  This unique structure, along with our proprietary chemistries, allow antisense therapies to be given by nearly all routes of administration, reaching nearly any part of the body.
  • SafeWith more than 12,000 patients treated with our antisense medicines to date, the Ionis antisense platform has been shown to be safe for patients across many disease areas.
Optimizing antisense with advances in medicinal chemistry 

Optimizing antisense through medicinal chemistry has been a central focus of our research efforts. By refining the foundational chemical structure of our antisense medicines, we have improved and continue to improve a number of characteristics of our platform:

  • Stability
  • Ability to penetrate certain tissues and cells
  • Specificity for its intended target
  • Binding strength (potency) for its intended target
  • Side effect profile
Making strides with Ligand-Conjugated Antisense (LICA) technology

Each generation of antisense technology builds upon the last. Ligand-Conjugated Antisense (LICA) technology, for instance, has allowed us to greatly improve the potency of our medicines in tissues we work in today and to potentially reach previously unreachable tissues throughout the body. LICA is a chemical modification that combines with our antisense therapies, targeting them to specific tissues, increasing the efficiency of drug uptake. LICA is leading to an emerging class of antisense medicines that can treat conditions, with both large and small patient populations, with less frequent and smaller doses.

Vector Smart Object

Each generation of antisense technology builds on the last. Our advances in medicinal chemistry are leading to medicines with:

  • Increased potency
  • Less frequent and smaller doses
  • More precise targeting to more tissues in the body
Administered through multiple routes of delivery

The elegant structure of single-stranded antisense therapies enables us to administer antisense therapies by nearly all routes of administration, reaching parts of the body that cause disease while minimizing drug exposure to other parts of the body, thereby providing improved safety and enabling us to expand the number of diseases we can treat.

Intraocular Intraocular
Inhalation_1 Oral
IV IV
Intradermal Intradermal
Intrathecal Intrathecal
Enema Enema
Intradermal_1 Subcutaneous
Inhalation Inhalation
Intellectual Property

We have a broad patent portfolio covering our products and technologies. The patent estate extends to our inventions in RNA-based drug discovery, technology advancements and to our specific drugs. It also encourages revenue-generating partnerships. Effectively protecting our intellectual property helps us maintain a competitive advantage in the field of antisense technology.

A perpetual and efficient innovation machine

Our platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed to treat a broad range of diseases.

Future-creating, antisense medicines

Our antisense technology has allowed us to create treatments that disrupt the disease process, may change its course and, we hope make a positive difference in patients' lives.