Advancing a diversified, sustainable pipeline
Our differentiated pipeline spans multiple modalities including RNA editing, splicing, RNA interference, and antisense silencing.
Our pipeline also includes clinical programs for diseases originating in muscle, the liver, and the central nervous system, and we are advancing preclinical programs across a variety of cell types throughout the body.
RNA EDITING
Program
Discovery / Preclinical
IND / CTA Enabling Studies
Clinical
Rights
Patient Population
(US & Europe)
(US & Europe)
RNA EDITING
RNA EDITING
WVE-006
SERPINA1 (AATD)
RestorAATion Clinical Program
GSK exclusive global license
200K
Multiple undisclosed Correction
100% global
>20K (multiple)
Multiple undisclosed Upregulation
100% global
>3M (multiple)
SPLICING
SPLICING
WVE-N531
Exon 53 (DMD)
FORWARD-53 Trial (Phase 2)
100% global
2.3K
Other exons (DMD)
100% global
Up to 18K
SILENCING: ANTISENSE
SILENCING: ANTISENSE
WVE-003
mHTT (HD)
SELECT-HD Trial (Phase 1b/2a)
Takeda 50:50 Option
25K Manifest (SNP3)
60K Pre-Manifest (SNP3)
SILENCING: RNAi
SILENCING: RNAi
INHBE
INHBE lead clinical candidate (Obesity and other metabolic disorders)
100% global
47M