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 | |
|---|---|---|---|---|---|---|
| ALS and FTD Variant-selective silencing |
WVE-004 C9orf72 |
Takeda 50:50 option |
||||
| Huntington's disease Allele-selective silencing |
WVE-003 mHTT SNP3 |
Takeda 50:50 option |
||||
| Spinocerebellar ataxia 3 Silencing | ATXN3 |
Takeda 50:50 option |
||||
| CNS diseases | Multiple† |
Takeda milestones & royalties |
||||
| Duchenne muscular dystrophy Exon skipping |
WVE-N531 Exon 53 |
100% global | ||||
| ADAR editing | Multiple | 100% global | ||||
| Alpha-1 antitrypsin deficiency ADAR editing | SERPINA1 | 100% global | ||||
| Retinal diseases |
USH2A and RhoP23H |
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
ALS and FTD
Variant-selective silencing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
WVE-004 C9orf72 |
|||
|
Partner Takeda 50:50 option |
|||
Huntington's disease
Allele-selective silencing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
WVE-003 mHTT SNP3 |
|||
|
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 |
|||
Duchenne muscular dystrophy
Exon skipping
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
WVE-N531 Exon 53 |
|||
|
Partner 100% global |
|||
ADAR editing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| Multiple | |||
|
Partner 100% global |
|||
HEPATIC
Alpha-1 antitrypsin deficiency
ADAR editing
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
| SERPINA1 | |||
|
Partner 100% global |
|||
OPHTHALMOLOGY
Retinal diseases
| Target | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
USH2A and RhoP23H |
|||
|
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
