For patients
A commitment
to deliver on the
promise of science
Our goal is to bring meaningful therapies to patients with serious genetic diseases.
This is our focus every single day. In pursuit of this goal, we employ unique insights and an innovative approach to traditional oligonucleotide development. As we work to advance our development programs to hopefully bring much-needed therapies to patients, our team shares a commitment to:
- Always put patients’ best interests first
- Listen to and partner with our communities and patient advocacy groups
- Move forward with a distinct sense of urgency
- Recognize there are high stakes to our work: patients and families are waiting for cures
Our commitment
Duchenne muscular dystrophy (DMD) is a fatal, X-linked genetic neuromuscular disorder that leads to progressive, irreversible loss of muscle function, including the heart and lungs. DMD is a serious genetic disease affecting boys worldwide; it is ultimately fatal.
impacts
~1/5,000
male newborns
around the world
~20K
new cases of DMD
annually worldwide
What happens in the body of a person with DMD?
DMD is a genetic disease caused by a mutation in the dystrophin gene which prevents the production of dystrophin protein, a vital component of healthy muscle function. Without dystrophin protein, muscle tissue is replaced by fat cells and muscles deteriorate over time.
How we’re tackling this
Our approach focuses on exon skipping, a mechanism that may allow for the body to produce shorter yet functional dystrophin protein.
Our molecule is designed to skip exon 53 within the dystrophin gene and contains chemistry modifications that may lead to more effective protein production.
~30K
people in the US are estimated
to be positive for HD and
exhibiting symptoms
~200K
people in the US are estimated
to be at risk of developing
the condition
What happens inside the body of a person with HD?
HD is caused by a mutation of the huntingtin (HTT) gene, which is critical for brain function. People with HD have an error in the huntingtin (HTT) gene, which tells our body how to make the huntingtin protein. All people have two copies of the huntingtin gene. A person only needs a genetic mutation in one of these copies to develop HD.
The mutation that causes HD is in a piece of the gene called the “CAG repeat.” The CAG repeat is made up of three “letters” of DNA that are scientifically referred to as Cytosine, Adenine and Guanine (CAG).
The genetic mutation that causes HD is a long sequence of the CAG repeat in the huntingtin gene:
- In a normal huntingtin gene, the CAG repeat occurs 10 to 35 times in a row.
- In the cells of a person with HD, it occurs 36 to more than 120 times.
This much longer segment instructs cells to make a longer version of the huntingtin protein, which results in deterioration of nerve cells in the brain. Some have described this as mHTT causing “serious mischief” in the brain.
Testing for HD involves measuring the CAG repeat length in both HD genes, using DNA obtained from a blood sample.
How we’re tackling this
We see an opportunity to use a genetic “GPS,” called a SNP, that will enable us to identify and target only the mutant gene. SNP stands for “single nucleotide polymorphism.” It is a scientific term for normal variations in the DNA between people.
Since SNPs are located in very specific spots in our DNA, they can act like a pin on a map, helping find the exact gene that causes a disease, preference or trait.
Our approach is to use these genetic “pins” to identify and knock down the protein that causes the disease in most patients, while still allowing the body to continue to produce the normal huntingtin protein, which is important for healthy brain function. We are the first company to target these genetic “pins” in clinical programs for HD.
This kind of approach is called “allele-specific gene silencing.”
“I have watched my cousin care for her mom over the past ten years of her slow decline due to HD. I grew up with my cousin, a large Irish family where the disease was never discussed, until now. Wave has given her hope that she will not burden her children with the same struggles.”
— Erin, Wave employee
Updates from Wave
Read our latest communications to the communities we serve.
- Wave Letter to HD Community (August 2023)
- Update on WVE-004 program in C9-ALS/FTD (May 2023)
- Next Steps for WVE-N531 in Duchenne Muscular Dystrophy (March 2023)
- Positive update on WVE-N531 in Duchenne (December 2022)
- Wave Letter to HD Community (September 2022)
Expanded Access Policy (EAP)
Wave Life Sciences (“Wave”) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Driven by a resolute sense of urgency, Wave is focused on research and development of treatments that target a broad range of genetically defined diseases, so that patients and families may realize a brighter future.
We believe the best, most appropriate way to deliver on our commitment—and drive the greatest benefit to the patient community as quickly as possible—is through our clinical trial programs. Please find information on Wave’s ongoing clinical trials here.
We recognize that not all patients will be able to enter these clinical trials, and in some cases, the patient’s physician may seek access to an investigational medicine. These requests are considered expanded access, or the use of an investigational new drug outside of a clinical trial for the diagnosis, monitoring, or treatment of patients with a life-threatening or seriously debilitating disease or condition who do not meet the enrollment criteria for clinical trials.
Given the early stage of our clinical development programs, our responsibility to “do no harm” to patients, and our steadfast commitment to advancing clinical understanding of our investigational medicines to benefit the most patients as quickly as possible, Wave does not currently offer an expanded access program for our investigational therapies. As more data on our investigational therapies become available, we will continue to review our policy on expanded access.
If you have any questions regarding Wave’s Expanded Access policy, please contact [email protected].
Our science
We have a cutting-edge scientific platform that is designed to target the underlying cause of disease.
Specifically, our focus is on nucleic acids—the body’s DNA and RNA.
In genetic diseases, mutations (or changes) in the body’s DNA can lead to complications with protein production. Proteins are considered the “workhorses” of our cells, and play an important role in our tissues and organs. For instance, proteins are important for movement and growth, for protection from harmful viruses and even for digestion.
When proteins are not produced properly by our body, they can’t do their jobs correctly. In some cases, a genetic mutation may result in too much of a specific protein. In other cases, the protein is absent, there is not enough, or it is mutated in some way. For example, it may be too long.
Our platform is designed to interact with the body’s genetic material using molecules called oligonucleotides.
Our platform is unique because we can build these molecules from the ground up. By controlling the characteristics of these oligonucleotides, we can potentially optimize what they can achieve, including where they go in the body, how they interact with other cellular functions, what they do once they reach the cell, and how they impact the body’s immune system.
We believe our unique insights and novel approach in rationally designed nucleic acids may enable us to develop meaningful therapies for patients with significant unmet needs.
A pledge to patients
Our patient advocacy team is responsible for ensuring that patient input is always at the forefront of our work. We know that it is critical to incorporate patient perspectives into the drug development process and our corporate culture. Our focus is on partnering with the community to better understand-and meet-patient needs by working with advocacy organizations and finding opportunities to listen to patients. To reach members of our team, contact [email protected].