For patients

A commitment
to deliver on the
promise of science

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 team has a unique connection to those affected by rare diseases. It’s a daily reminder that our efforts could directly impact the lives of so many extraordinary people. These individuals are not only patients but families, communities."
Melanie, Wave employee

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.

Our commitment

What is Huntington’s disease?

Huntington’s disease (HD) is a devastating genetic disorder that deeply impacts both patients and their loved ones. HD causes nerve cells in the brain to deteriorate over time, affecting thinking ability, emotions and movement. It is ultimately fatal.

~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

What is amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS) is a disease that causes ongoing deterioration of motor neurons in the brain and spinal cord. This leads to the inability to initiate or control muscle movement. People with ALS may lose the ability to speak, eat, move and breathe. It is ultimately fatal.

~20K

people in the US
have ALS

2.5yr

median survival rate

What happens in the body of a person with ALS?

ALS can be caused by mutations in the C9orf72 gene, which provides instructions for making protein found in various tissues, including nerve cells in the brain and muscles.

A normal (healthy) C9orf72 gene contains 2-23 hexanucleotide repeats, which are sections within the gene. In a person with ALS, the genetic mutation contains hundreds to thousands of hexanucleotide repeats. This much longer segment causes the formation of mutant proteins that gather in brain tissue.

How we’re tackling this

Our approach aims to knock down the process that results in the harmful proteins.

“As a 10-year-old, when my father surrendered his body to ALS, there wasn’t much I could do except raise money. Almost 15 years later, it is very exciting to be on the Wave team personally contributing to meaningful research.”

— Chandlar, Wave employee

What is frontotemporal dementia?

Frontotemporal dementia (FTD) is a disease that causes ongoing nerve cell loss in the brain’s frontal lobes and temporal lobes. This leads to personality and behavioral changes, as well as the gradual impairment of language skills.

~55K

people in the US
have FTD

2nd

most common form of
early onset dementia after
Alzheimer’s disease in people
under the age of 65

What happens in the body of a person with FTD?

FTD can be caused by mutations in the C9orf72 gene, which provides instructions for making protein found in various tissues, including nerve cells in the brain and muscles.

A normal (healthy) C9orf72 gene contains 2-23 hexanucleotide repeats, which are sections within the gene. In a person with FTD, the genetic mutation contains hundreds to thousands of hexanucleotide repeats. This much longer segment causes the formation of mutant proteins that gather in brain tissue.

How we’re tackling this

Our approach aims to knock down the process that results in the harmful proteins.

The clinical trials process tests potential treatments in patients to see whether they are safe and effective and should be approved for use in the general population. A treatment could be a medicine, medical device, vaccine or even gene therapy. Clinical trials are an important part of the development of new products and are generally required by regulatory authorities before any new treatment can be brought to market.

Our clinical trials in

Huntington’s disease

Trial
Recruiting

We are currently recruiting adults with early manifest Huntington’s disease (HD) who carry a SNP at the rs362307 (“SNP1”) location for a Phase 1b/2a clinical trial.

Learn more about Wave trials for HD at clinicaltrials.gov

Trial
Recruiting

We are currently recruiting adults with early manifest Huntington’s disease (HD) who carry a SNP at the rs362331 (“SNP2”) location for a Phase 1b/2a clinical trial.

Learn more about Wave trials for HD at clinicaltrials.gov

Additional Programs in Development

Wave is advancing its C9orf72 preclinical program to potentially treat amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

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. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM™, the company’s proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future.

Expanded access is 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. Currently, Wave does not offer an expanded access program for our investigational therapies. We are committed to providing patients with access to our therapies at the appropriate time with a focus on minimizing any potential risks to patients.

At this time, we believe the most appropriate way to deliver on this commitment is through participation in our clinical trials. We are working to advance our clinical programs as quickly as possible so that we may potentially offer access to tested and approved therapies. Please find information on Wave’s ongoing clinical trials here clinicaltrials.gov >

We recognize that not all patients will be able to enter these clinical trials, and as more information and clinical data on the safety and efficacy of our investigational therapies become available, we will review and potentially modify our policy on expanded access.

If you have any questions regarding Wave’s Expanded Access policy, please contact [email protected]. We anticipate acknowledging receipt of inquiries sent to this email within five business days.

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].

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].