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 | 100% global | - | ||||
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) | ||||
SCA3 (ATXN3) |
Takeda 50:50 Option |
8K | ||||
SILENCING: RNAi | ||||||
Multiple | 100% global | - |
Through GSK collaboration, Wave can advance up to three collaboration 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
WVE-006
SERPINA1 (AATD)
Discovery | Preclinical | Clinical | Rights |
---|---|---|---|
GSK exclusiveglobal license | |||
Patient Population (US & Europe) 200K |
Multiple
Discovery | Preclinical | Clinical | Rights |
---|---|---|---|
100% global | |||
|
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) |
SCA3 (ATXN3)
Discovery | Preclinical | Clinical | Rights |
---|---|---|---|
Takeda 50:50 Option | |||
Patient Population (US & Europe) 8K |
Multiple
Discovery | Preclinical | Clinical | Rights |
---|---|---|---|
100% global | |||
|
Through GSK collaboration, Wave can advance up to three collaboration 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