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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.

Program
Discovery
Preclinical
Clinical
Rights
Patient Population
(US & Europe)

RNA EDITING

WVE-006

SERPINA1 (AATD)
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RestorAATion Clinical Program

GSK exclusive global license

200K

Multiple undisclosed Correction
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100% global

>20K (multiple)

Multiple undisclosed Upregulation
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100% global

>3M (multiple)

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

WVE-003

mHTT (HD)
SELECT-HD Trial (Phase 1b/2a)

Takeda 50:50 Option

25K Manifest (SNP3)
60K Pre-Manifest (SNP3)

SILENCING: RNAi

INHBE

Obesity and other metabolic disorders

100% global

47M