Tapping into a world of potential

Tapping into a world of potential

Nucleic acids are molecules that allow genetic information to pass from one generation to the next, for example DNA and RNA.

Nucleic acid therapeutics are chemically synthesized oligonucleotides that aim to rectify problems within genetic code, most often mutations within genes.

Made of chemically modified short-length RNA or DNA strands, these oligonucleotides interact with the complementary target messenger RNA within our genes to change the production of proteins that cause disease. They hold the potential to treat many diseases that have historically been difficult to address.

Unlocking the future of medicine

To help stabilize oligonucleotides from degradation, their natural phosphate (PO) backbone is frequently modified to phosphorothioates (PS).

Specifically, one of the non-bridging oxygen atoms in the phosphate is replaced with a sulfur atom at each phosphate linkage, creating a chiral center with three-dimensional properties at each link.

In traditional synthesis, these chiral configurations are not purposefully designed or controlled, creating mixtures of, in many cases, over half a million different molecules, each having varied three-dimensional atomic arrangements.

This leads to potentially differing pharmacologic properties, resulting in inconsistent therapeutic effects and unintended off-target effects.

Rational design enables the optimization of critical constructs into a defined and more consistent profile, opening up the potential for safer, more effective medicines.

Creating a new
class of medicines

Wave’s proprietary chemistry platform enables the precise design, optimization and production of stereopure oligonucleotides. Both stereochemistry and chemical modification can be consistently controlled, with atoms precisely arranged in three-dimensional orientations at each linkage.

This may enable:

Optimized pharmacological properties:

  • Greater stability
  • Improved activity and selectivity
  • Longer duration of effect
  • Enhanced clearance

Unique versatility: broad applicability across modalities, including:

  • Antisense
  • Exon skipping
  • Splice correction
  • RNAi
  • RNA-guided gene editing
  • MicroRNA

The potential to make medicines with an improved benefit-risk profile to treat many diseases for which there are currently few or no treatment options

This may enable:

Optimized pharmacological properties:

  • Greater stability
  • Improved activity and selectivity
  • Longer duration of effect
  • Enhanced clearance

Unique versatility: broad applicability across modalities, including:

  • Antisense
  • Exon skipping
  • Splice correction
  • RNAi
  • RNA-guided gene editing
  • MicroRNA

The potential to make medicines with an improved benefit-risk profile to treat many diseases for which there are currently few or no treatment options

This may enable:

Optimized pharmacological properties:

  • Greater stability
  • Improved activity and selectivity
  • Longer duration of effect
  • Enhanced clearance

Unique versatility: broad applicability across modalities, including:

  • Antisense
  • Exon skipping
  • Splice correction
  • RNAi
  • RNA-guided gene editing
  • MicroRNA

The potential to make medicines with an improved benefit-risk profile to treat many diseases for which there are currently few or no treatment options

How it works:
built from the ground up

Identify

Target Sequence

Apply

Antisense RNAi Exon Skipping

Control

Wave Chemistry Platform

Design

Wave Rational Design

Optimize

Stability Activity Immune Specificity

Validate

Identify

Target Sequence

Apply

Antisense

RNAi

Exon Skipping

Control

Wave Chemistry
Platform

Design

Wave Rational
Design

Optimize

Stability

Activity

Immune

Specificity

Validate

Wave Optimized Isomers

Just one of the millions of reasons to do better

We are exploring the application of our technology in many serious genetic disease areas in which there are unacceptably few or no treatment options.

Learn more about our lead programs