Our pipeline:
multiple modalities and novel targets
We are developing disease-modifying therapies for several genetic conditions with a high degree of unmet need. While our initial focus is in neurology, our stereopure chemistry platform may unlock the potential for transformative therapies in many therapeutic areas.
Our diverse discovery programs aim to explore the potential of our platform across a broad range of serious genetic diseases.
|
Therapeutic Area/Modality |
Target | Discovery | Preclinical | Clinical | Partner | |
|---|---|---|---|---|---|---|
| Huntington's disease Allele-selective silencing |
WVE-120101 mHTT SNP1 |
Takeda 50:50 option |
||||
|
WVE-120102 mHTT SNP2 |
Takeda 50:50 option |
|||||
| mHTT SNP3 |
Takeda 50:50 option |
|||||
| ALS and FTD Allele-selective silencing | C9orf72 |
Takeda 50:50 option |
||||
| Spinocerebellar ataxia 3 Silencing | ATXN3 |
Takeda 50:50 option |
||||
| CNS diseases | Multiple† |
Takeda milestones & royalties |
||||
| Retinal diseases |
USH2A and RhoP23H |
100% global | ||||
| Metabolic liver diseases Silencing | Multiple |
Pfizer milestones & royalties |
||||
| ADAR RNA-editing | Multiple | 100% global | ||||
†During a four-year term, Wave and Takeda may collaborate on up to six preclinical targets at any one time.
ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system; OLE: Open-label extension
CNS
Huntington's disease
Allele-selective silencing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
WVE-120101 mHTT SNP1 |
|||
|
Partner Takeda 50:50 option |
|||
|
WVE-120102 mHTT SNP2 |
|||
|
Partner Takeda 50:50 option |
|||
| mHTT SNP3 | |||
|
Partner Takeda 50:50 option |
|||
ALS and FTD
Allele-selective silencing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| C9orf72 | |||
|
Partner Takeda 50:50 option |
|||
Spinocerebellar ataxia 3
Silencing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| ATXN3 | |||
|
Partner Takeda 50:50 option |
|||
CNS diseases
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| Multiple† | |||
|
Partner Takeda milestones & royalties |
|||
OPHTHALMOLOGY
Retinal diseases
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
USH2A and RhoP23H |
|||
|
Partner 100% global |
|||
HEPATIC
Metabolic liver diseases
Silencing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| Multiple | |||
|
Partner Pfizer milestones & royalties |
|||
OTHER
ADAR
RNA-editing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| Multiple | |||
|
Partner 100% global |
|||
†During a four-year term, Wave and Takeda may collaborate on up to six preclinical targets at any one time.
ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system; OLE: Open-label extension
Find out about Wave