Aperture

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.

Thesis

ATTR-CM is the rare disease that stopped being rare the moment nuclear scintigraphy and tafamidis existed. The market is now a three-way fight — stabilizer (Pfizer tafamidis, BridgeBio acoramidis) versus silencer (Alnylam vutrisiran, Ionis/AZ eplontersen) — with gene editing sitting one readout away from rewriting the peak. For Corp Dev this is a sequencing and share-shift problem, not an epidemiology discovery problem.

wtATTR global (dir.)

200–300k

vATTR global (dir.)

10–40k

HFpEF pocket rate

up to ~13%

Approved CM class

Stabilizer + silencer

Tafamidis LoE

~2028

Next binary

In vivo editing

So-what for Corp Dev

  1. 01Diagnosis is the volume lever. Prevalence in selected HFpEF / AS / LVH pockets is 10–15%, not the historical 'rare' base rate.
  2. 02Tafamidis LoE around 2028 is the cliff. Acoramidis and silencers are fighting to own the post-LoE stack.
  3. 03Mixed phenotype (~25–33%) means the neuro franchise and the cardio franchise are the same patient more often than models assume.
  4. 04NTLA-2001-class editors are the only assets that can turn this back into a one-time market.

Residual unmet need

  • Late diagnosis

    Years of HFpEF before a PYP scan. Every month untreated is irreversible infiltration.

  • Advanced NAC / NYHA IV

    Stabilizers and silencers slow; they do not empty the heart. Late presenters have little reversible substrate.

  • Hereditary mixed disease

    Val50Met and others need a product story that is honest about polyneuropathy plus cardiomyopathy.

Marketed share (dir.)

  • Vyndaqel / Vyndamax54%
  • Amvuttra18%
  • Attruby16%
  • Wainua8%
Algorithm

Late pipeline

  • Nexiguran ziclumeran (NTLA-2001)

    Intellia / Regeneron · Phase 3 · Gene editing

Full pipeline

Asset dives

Case study with selected pricing and peak-sales fields held back.