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Hereditary Transthyretin Amyloidosis: one US-listed biotech with a clinical-stage programme

OphthalmologyUncontestedData as of 2026-09-04

Competitors1in the clinic
Median market cap$9.7Buniverse median $1.20B
Allocated capital$1.1B$9.7B competing
Phase 3 or filed10 mid, 0 early
Pure plays0this indication only
Capital rank144/299crowding rank 158

Hereditary Transthyretin Amyloidosis is uncontested. Ionis Pharmaceuticals (IONS) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with Eplontersen in phase 3.

There is no public comparable to price it against. IONS spreads its $9.7B market cap across 9 indications, so $1.1B of it is allocated here.

The one programme here is in late-stage development, with 1 dated catalyst in the next 12 months.

$1.1B of allocated capital sits in Hereditary Transthyretin Amyloidosis, ranking it 144th of 299 indications by the money committed to it.

Companies developing drugs for Hereditary Transthyretin Amyloidosis 1

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
IONSIonis PharmaceuticalsEplontersenPhase 31$9.7BNo9 22 Sep 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile.

Why Hereditary Transthyretin Amyloidosis counts as uncontested

Uncontested. One US-listed developer in the size band is running a clinical programme here, so there is no public comparable to price it against.

The map splits indications on two folds: six or more competitors counts as crowded, and a median company above $1.20B counts as richly priced. Hereditary Transthyretin Amyloidosis has one competitor and a median of $9.7B.

Ophthalmology

Ophthalmology holds $17.0B of the map's capital, 2.5% of the total, spread across 9 indications and 13 companies. 6 of those indications have more than one company competing.

See every Ophthalmology indication on the map

Related indications in Ophthalmology

Diabetic Macular Edema4 companies · $1.3B · median $613MOrphan cornerMacular Edema1 company · $758M · median $2.3BUncontestedLeber Congenital Amaurosis2 companies · $600M · median $300MOrphan cornerX-Linked Retinitis Pigmentosa1 company · $192M · median $768MUncontestedDry Eye Disease3 companies · $2.0B · median $706MOrphan cornerWet Age-related Macular Degeneration4 companies · $3.5B · median $1.5BConcentrated betStargardt Disease2 companies · $3.6B · median $3.7BConcentrated betGeographic Atrophy3 companies · $4.0B · median $918MOrphan corner

Hereditary Transthyretin Amyloidosis pipeline questions

Which biotech companies are developing drugs for Hereditary Transthyretin Amyloidosis?

Ionis Pharmaceuticals (IONS, Eplontersen, Phase 3). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Hereditary Transthyretin Amyloidosis.

How advanced is the Hereditary Transthyretin Amyloidosis pipeline?

Of the 1 clinical-stage programme in Hereditary Transthyretin Amyloidosis, 1 is in Phase 3 or filed, 0 are in mid-stage development and 0 are in early-stage development.

Is anyone else developing a drug for Hereditary Transthyretin Amyloidosis?

No. IONS is the only US-listed company in the $100M to $20B market cap band with a clinical-stage programme in Hereditary Transthyretin Amyloidosis, which is why the indication is classified as uncontested.

When is the next Hereditary Transthyretin Amyloidosis catalyst?

The next dated catalyst in Hereditary Transthyretin Amyloidosis is IONS on 22 Sep 2026. 1 dated event across the field fall in the next 12 months.

How this page is built

  • Universe is US-listed drug developers with a $100M to $20B market capitalisation, identified by SEC SIC code 2833, 2834 or 2836 or by a pharmaceutical or biotechnology industry classification, less a reviewed exclusion list of contract research organisations, device and diagnostics makers and diversified generics manufacturers.
  • An asset attributed to three or more different companies is treated as shared standard of care rather than any one company’s programme and excluded. Pembrolizumab alone appears under 21 tickers in raw trial data.
  • A programme counts only where at least one industry-sponsored trial supports it, so investigator-initiated academic studies of third-party drugs are excluded.

Allocated capital divides each company’s market capitalisation evenly across the indications it is running clinical programmes in, so every dollar of the universe is counted exactly once. Total market capitalisation competing counts a company in each of its indications and therefore sums to more than the universe.

Third-party market size estimates are not used anywhere on this page. Approved incumbents are excluded from competitor counts, and companies above $20B are outside the universe by construction.

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