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) | |
|---|---|---|---|---|---|---|
| RNA EDITING | ||||||
|
WVE-006
SERPINA1 (AATD)
 |
GSK exclusiveglobal license | 200K | ||||
|
Multiple undisclosedCorrection
|
100% global | >20K (multiple) | ||||
|
Multiple undisclosedUpregulation
 |
100% global | >3M (multiple) | ||||
| SPLICING | ||||||
| WVE-N531 Exon 53 (DMD) | 100% global | 2.3K | ||||
| Other exons (DMD) | 100% global | Up to 18K | ||||
| SILENCING: ANTISENSE | ||||||
| WVE-003 mHTT (HD) |
Takeda 50:50 Option |
25K Manifest (SNP3)60K Pre-Manifest (SNP3) | ||||
| SILENCING: siRNA | ||||||
| INHBE* (metabolic disorders, including obesity) | 100% global | 47M | ||||
*Through GSK collaboration, Wave can advance up to three collaboration programs (the first of which is INHBE) and GSK can advance up to eight collaboration programs.
AATD: Alpha-1 antitrypsin deficiency; DMD: Duchenne muscular dystrophy; HD: Huntington's disease Editing for correction Editing for upregulation
WVE-006
SERPINA1 (AATD)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| GSK exclusiveglobal license | |||
|
Patient Population (US & Europe) 200K |
|||
Multiple undisclosedCorrection
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
Patient Population (US & Europe) >20K (multiple) |
|||
Multiple undisclosedUpregulation
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
Patient Population (US & Europe) >3M (multiple) |
|||
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 |
|||
WVE-003
mHTT (HD)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| Takeda 50:50 Option | |||
|
Patient Population (US & Europe) 25K Manifest (SNP3); 60K Pre-Manifest (SNP3) |
|||
INHBE* (metabolic disorders, including obesity)
| Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|
| 100% global | |||
|
Patient Population (US & Europe) 47M |
|||
*Through GSK collaboration, Wave can advance up to three collaboration programs (the first of which is INHBE) and GSK can advance up to eight collaboration programs.
AATD: Alpha-1 antitrypsin deficiency; DMD: Duchenne muscular dystrophy; HD: Huntington's disease Editing for correction Editing for upregulation
