Pipeline

Pipeline

Akcea is advancing a mature pipeline to address the unmet needs and complex medical challenges of patients with serious and rare diseases using antisense technology.

Transthyretin amyloidosis (ATTR)

Pre-clinical
Phase 1
Phase 2
Phase 3
Registration
AKCEA-TTR-LRx
ATTR Amyloidosis (ATTR)
Phase 3

Cardiometabolic lipid disorders

Volanesorsen
Pre-clinical
Phase 1
Phase 2
Phase 3
Approved in the EU
Familial Chylomicronemia Syndrome (FCS)
Approved in the EU
Pelacarsen (AKCEA-APO(a)-LRx)
Pre-clinical
Phase 1
Phase 2
Phase 3
Registration
Hyperlipoproteinemia(a) with CV risk
Phase 3
Vupanorsen (AKCEA-ANGPTL3-LRx)
Cardiovascular and Metabolic diseases
Phase 2
AKCEA-APOCIII-LRx
Hypertriglyceridemia with CV risk
Phase 2
IONIS

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