Our pipeline:
multiple modalities and novel targets
We are committed to delivering life-changing RNA medicines so that patients and families can realize a brighter future.
Our pipeline is differentiated and diversified, spanning multiple modalities including RNA editing, splicing and silencing, as well as several disease areas.
| Program | Discovery | Preclinical | Clinical | Rights | Patient Population(US & Europe) | |
|---|---|---|---|---|---|---|
| SPLICING | ||||||
| WVE-N531 Exon 53 (DMD) | 100% global | 2.3K | ||||
| Other exons (DMD) | 100% global | Up to 18K | ||||
| RNA EDITING | ||||||
| WVE-006 SERPINA1 (AATD) | GSK exclusiveglobal license | 200K | ||||
| Multiple | 100% global | - | ||||
| SILENCING: ANTISENSE | ||||||
| WVE-003 mHTT (HD) |
Takeda 50:50 Option |
25K Manifest (SNP3)60K Pre-Manifest (SNP3) | ||||
| SCA3 (ATXN3) |
Takeda 50:50 Option |
8K | ||||
| SILENCING: RNAi | ||||||
| Multiple | 100% global | - | ||||
Through GSK collaboration, Wave can advance up to three programs and GSK can advance up to eight collaboration programs
AATD: Alpha-1 antitrypsin deficiency; DMD: Duchenne muscular dystrophy; HD: Huntington's disease; SCA3: Spinocerebellar ataxia 3
Splicing
WVE-N531
Exon 53 (DMD)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
Patient Population (US & Europe) 2.3K |
|||
Other exons (DMD)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
Patient Population (US & Europe) Up to 18K |
|||
RNA Editing
WVE-006
SERPINA1 (AATD)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| GSK exclusiveglobal license | |||
|
Patient Population (US & Europe) 200K |
|||
Multiple
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
|
|||
SILENCING: ANTISENSE
WVE-003
mHTT (HD)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| Takeda 50:50 Option | |||
|
Patient Population (US & Europe) 25K Manifest (SNP3); 60K Pre-Manifest (SNP3) |
|||
SCA3 (ATXN3)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| Takeda 50:50 Option | |||
|
Patient Population (US & Europe) 8K |
|||
SILENCING: RNAi
Multiple
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
|
|||
Through GSK collaboration, Wave can advance up to three programs and GSK can advance up to eight collaboration programs
AATD: Alpha-1 antitrypsin deficiency; DMD: Duchenne muscular dystrophy; HD: Huntington's disease; SCA3: Spinocerebellar ataxia 3
