Cardiology · Rare-to-common · teaser
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.
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| Asset | Sponsor | Phase | Modality | Population | Signal |
|---|---|---|---|---|---|
| Nexiguran ziclumeran (NTLA-2001) In vivo CRISPR TTR knockout | Intellia / Regeneron | Phase 3 | Gene editing | ATTR-CM / ATTR-PN | Deep, durable TTR cut. The one-time question. |
| Next-wave silencers TTR knockdown | Several | Phase 2 | siRNA | ATTR-CM | held |