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Sickle Cell Disease: 6 biotech companies competing in the clinic

Rare Genetic & MetabolicGold rushData as of 2026-09-04

Competitors6in the clinic
Median market cap$2.5Buniverse median $1.20B
Allocated capital$4.8B$15.1B competing
Phase 3 or filed21 mid, 3 early
Pure plays2this indication only
Capital rank38/299crowding rank 27

Sickle Cell Disease is both crowded and richly priced: 6 companies are in the clinic and the median one is worth $2.5B, against a universe median of $1.20B.

Crowded fields usually trade at a discount per company, so a premium in a field this deep says the market is pricing the size of the prize above the cost of the competition.

2 of the 6 are in Phase 3 or filed, and 5 dated catalysts fall in the next 12 months, so this ranking is likely to move.

$4.8B of allocated capital sits in Sickle Cell Disease, 38th of 299 indications, and 2 of the 6 companies are pure plays that trade on this indication alone.

Companies developing drugs for Sickle Cell Disease 6

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
TVTXTravere TherapeuticsL-citrullinePhase 31$6.3BNo4 31 Dec 2026
AGIOAgios PharmaceuticalsMitapivatPhase 33$2.0BNo6 31 Dec 2026
FULCFulcrum TherapeuticsPociredirPhase 21$293MYes1 30 Jun 2027
IRONDisc MedicineDISC-3405Phase 11$3.0BNo5 9 Sep 2026
BEAMBeam TherapeuticsBEAM-101Phase 11$3.0BNo2 8 Sep 2026
EDITEditas MedicineEDIT-301Phase 11$473MYes1 31 Dec 2027

Sorted by phase, then by market capitalisation. Ticker links open the company profile. TVTX holds 42% of the $15.1B competing in this indication.

Why Sickle Cell Disease is a gold rush

Crowded and richly priced: six or more competitors, and the median one is worth more than the median small or mid cap biotech.

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. Sickle Cell Disease has 6 competitors and a median of $2.5B.

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

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Sickle Cell Disease pipeline questions

Which biotech companies are developing drugs for Sickle Cell Disease?

6 US-listed companies in the $100M to $20B market cap band run clinical programmes in Sickle Cell Disease. The largest and most advanced are Travere Therapeutics (TVTX, L-citrulline, Phase 3); Agios Pharmaceuticals (AGIO, Mitapivat, Phase 3); Fulcrum Therapeutics (FULC, Pociredir, Phase 2); Disc Medicine (IRON, DISC-3405, Phase 1); Beam Therapeutics (BEAM, BEAM-101, Phase 1). The full list of 6 is in the table on this page.

How advanced is the Sickle Cell Disease pipeline?

Of the 6 clinical-stage programmes in Sickle Cell Disease, 2 are in Phase 3 or filed, 1 is in mid-stage development and 3 are in early-stage development.

How much is the typical Sickle Cell Disease biotech company worth?

The median company developing a drug for Sickle Cell Disease is worth $2.5B, against a median of $1.20B across the whole universe. $4.8B of allocated capital sits in the indication, ranking it 38th of 299.

When is the next Sickle Cell Disease catalyst?

The next dated catalyst in Sickle Cell Disease is BEAM on 8 Sep 2026. 5 dated events 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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