Cardiology · Rare-to-common · teaser

ATTR cardiomyopathy

Updated August 2026 · TTR (wild-type + hereditary)

Teaser case. Selected price and peak-sales fields are held for a paid Brief — the rest is the method.

← Pipeline

Intellia / Regeneron · Gene editing · Phase 3

Nexiguran ziclumeran

Thesis

One-time TTR knockout. If durability and safety hold, every chronic ATTR peak needs a haircut.

Probability of success

held

  • Durable knockdown in pivotal CM High
  • Off-target / liver class risk High

Time to market

held

  • Available on Brief

Peak revenue (US+EU+JP dir.)

held

Peak build

  • Peak build held

What kills the number

  • A single safety event in editing freezes the category.