ATTR amyloidosis

ATTR · Rare Disease · 3 drugs · 5 indications

Transthyretin amyloid deposits. Includes hereditary and wild-type.

Competitive Landscape (3 drugs)

Drug	Company	Mechanism	Modality	Route	Stage
Nexiguran ziclumeran	REGN	TTR gene editing (CRISPR/Cas9)	In vivo CRISPR gene editing	IV	PHASE3
Amvuttra	ALNY	RNAi (RNA Interference)	siRNA	SC (quarterly)	APPROVED
Wainua	AZN	TTR silencer	Antisense oligonucleotide	SC	APPROVED

Indications (5)

hATTR Amyloidosis (Cardiomyopathy)

Amvuttra APPROVEDWainua PHASE3

Hereditary transthyretin amyloidosis polyneuropathy

Wainua APPROVED

hATTR Amyloidosis (Polyneuropathy)

Amvuttra APPROVED

ATTR cardiomyopathy (ATTR-CM)

Hereditary ATTR polyneuropathy (ATTRv-PN)

Upcoming Catalysts

Wainua - ATTR-CM - Ph3 - Topline (CARDIO-TTRansform)CLINICAL

AZNH2 2026

Data from Supabase · Updated 2026-03-24