Recent Updates
Recently added Catalysts

Thalassemia: one US-listed biotech with a clinical-stage programme

Rare Genetic & MetabolicUncontestedData as of 2026-09-04

Competitors1in the clinic
Median market cap$2.0Buniverse median $1.20B
Allocated capital$330M$2.0B competing
Phase 3 or filed01 mid, 0 early
Pure plays0this indication only
Capital rank233/299crowding rank 236

Thalassemia is uncontested. Agios Pharmaceuticals (AGIO) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with AG-348 in phase 2.

There is no public comparable to price it against. AGIO spreads its $2.0B market cap across 6 indications, so $330M of it is allocated here.

Nothing here has reached Phase 3: 1 programme in mid stage and 0 in early stage, so the field is still several years from a first approval.

$330M of allocated capital sits in Thalassemia, ranking it 233rd of 299 indications by the money committed to it.

Companies developing drugs for Thalassemia 1

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
AGIOAgios PharmaceuticalsAG-348Phase 21$2.0BNo6 31 Dec 2026

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

Why Thalassemia 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. Thalassemia has one competitor and a median of $2.0B.

Rare Genetic & Metabolic

Rare Genetic & Metabolic holds $103B of the map's capital, 15.3% of the total, spread across 32 indications and 48 companies. 10 of those indications have more than one company competing.

See every Rare Genetic & Metabolic indication on the map

Related indications in Rare Genetic & Metabolic

Transfusion Dependent Beta-thalassemia1 company · $330M · median $2.0BUncontestedPyruvate Kinase Deficiency1 company · $330M · median $2.0BUncontestedEpidermolysis Bullosa Dystrophica1 company · $339M · median $339MUncontestedMucopolysaccharidosis Type II (MPS II)1 company · $304M · median $607MUncontestedGuillain-Barré Syndrome1 company · $459M · median $918MUncontestedParoxysmal Nocturnal Hemoglobinuria1 company · $461M · median $1.4BUncontestedConstipation Predominant Irritable Bowel Syndrome1 company · $462M · median $924MUncontestedOsteogenesis Imperfecta Type IV1 company · $488M · median $1.5BUncontested

Thalassemia pipeline questions

Which biotech companies are developing drugs for Thalassemia?

Agios Pharmaceuticals (AGIO, AG-348, Phase 2). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Thalassemia.

How advanced is the Thalassemia pipeline?

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

Is anyone else developing a drug for Thalassemia?

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

When is the next Thalassemia catalyst?

The next dated catalyst in Thalassemia is AGIO on 31 Dec 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.

Back to the disease map