Building the
optimal medicine
We are leading a new era of precision medicine designed to deliver safer, more effective treatment options.
Our science represents a groundbreaking approach in nucleic acid therapeutic technology, with the potential to transform the treatment of rare genetic diseases.
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 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
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Identify
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Target Sequence
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Apply
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Antisense
RNAi
Exon Skipping
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Control
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Wave Chemistry Platform
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Design
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Wave Rational Design
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Optimize
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Stability
Activity
Immune
Specificity
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Validate
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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