United States

We are leading a new era of precision medicine in which rationally designed oligonucleotides are the key to delivering safer, more effective medicines. These include potentially transformational therapies for conditions where there are currently few or no treatment options. We aspire to help create a world in which every patient with a genetic disease will have access to life-changing treatments in their lifetime.

Central Nervous System (CNS)

Genetic Diseases

Liver Diseases

Musculoskeletal Diseases

Wave Life Sciences

Duchenne muscular dystrophy

WVE-N531:Our approach focuses on exon skipping, a mechanism that may allow for the production of truncated (or shortened) functional dystrophin protein. Specifically, we have developed an investigational oligonucleotide therapeutic designed to skip exon 53 and restore the mRNA reading frame. This candidate incorporates Wave’s proprietary PN chemistry, which has been shown clinically and preclinically to improve pharmacological properties of exon skipping oligonucleotides.

WVE-N531

Obesity

WVE-007  [GalNAc-small interfering RNA (siRNA)] INHBE Program

Alpha-1 Antitrypsin Deficiency

WVE-006

Huntington's disease

WVE-003 is designed to selectively target the mutant allele of the huntingtin (mHTT) gene, while leaving the wild-type (healthy) HTT (wtHTT) protein relatively intact. Wave’s approach to HD is guided by the recognition that, in addition to a gain of function of the mHTT protein, people with this disease have less wtHTT protein, leaving them with a smaller protective reservoir of healthy protein compared to unaffected individuals.

WVE-003

Amyotrophic lateral sclerosis

Frontotemporal dementia with GRN mutations

WVE-004

Our programs target two disease-associated single nucleotide polymorphisms (“SNPs”) within the huntingtin gene: rs362307 (“mHTT SNP-1”) and rs362331 (“mHTT SNP-2”). SNPs are naturally occurring variations within a given genetic sequence. In certain instances, SNPs can be used to distinguish between two related copies of a gene, where only one is responsible for causing production of a defective protein that causes disease. It has been shown that by targeting mHTT SNP-1 and mHTT SNP-2, the production of disease-causing proteins associated with HD can be reduced. This has the potential to provide treatment for up to 70% of people with HD.

Wave Life Sciences

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