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Alpha-1 Antitrypsin Deficiency: 5 biotech companies with clinical-stage programmes

Rare Genetic & MetabolicConcentrated betData as of 2026-09-04

Competitors5in the clinic
Median market cap$3.0Buniverse median $1.20B
Allocated capital$7.6B$29.9B competing
Phase 3 or filed10 mid, 4 early
Pure plays0this indication only
Capital rank23/299crowding rank 32

Alpha-1 Antitrypsin Deficiency is a concentrated bet: only 5 US-listed developers are in the clinic, and the median one is worth $3.0B against a $1.20B universe median.

Scarcity is doing the work in that number. A thin field means an approval is shared with fewer rivals, and the market pays a premium for that position.

1 of the 5 is in Phase 3 or filed, and 4 dated catalysts fall in the next 12 months, so this ranking is likely to move.

$7.6B of allocated capital sits in Alpha-1 Antitrypsin Deficiency, ranking it 23rd of 299 indications by the money committed to it.

Companies developing drugs for Alpha-1 Antitrypsin Deficiency 5

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
GRFSGrifols, S.A. Sponsored ADRAlpha-1 MPPhase 33$2.1BNo2 31 Dec 2026
BMRNBioMarin PharmaceuticalBMN 349Phase 11$12.9BNo5No dated event
KRYSKrystal BiotechKB408Phase 11$11.0BNo5 31 Dec 2026
BEAMBeam TherapeuticsBEAM-302Phase 11$3.0BNo2 8 Sep 2026
WVEWave Life SciencesWVE-006Phase 11$904MNo3 31 Dec 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile. BMRN holds 43% of the $29.9B competing in this indication.

Why Alpha-1 Antitrypsin Deficiency is a concentrated bet

Few competitors, each one large. The market pays a scarcity premium to compete here.

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. Alpha-1 Antitrypsin Deficiency has 5 competitors and a median of $3.0B.

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.

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Alpha-1 Antitrypsin Deficiency pipeline questions

Which biotech companies are developing drugs for Alpha-1 Antitrypsin Deficiency?

Grifols, S.A. Sponsored ADR (GRFS, Alpha-1 MP, Phase 3); BioMarin Pharmaceutical (BMRN, BMN 349, Phase 1); Krystal Biotech (KRYS, KB408, Phase 1); Beam Therapeutics (BEAM, BEAM-302, Phase 1); Wave Life Sciences (WVE, WVE-006, Phase 1). Those are the 5 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Alpha-1 Antitrypsin Deficiency.

How advanced is the Alpha-1 Antitrypsin Deficiency pipeline?

Of the 5 clinical-stage programmes in Alpha-1 Antitrypsin Deficiency, 1 is in Phase 3 or filed, 0 are in mid-stage development and 4 are in early-stage development.

How much is the typical Alpha-1 Antitrypsin Deficiency biotech company worth?

The median company developing a drug for Alpha-1 Antitrypsin Deficiency is worth $3.0B, against a median of $1.20B across the whole universe. $7.6B of allocated capital sits in the indication, ranking it 23rd of 299.

When is the next Alpha-1 Antitrypsin Deficiency catalyst?

The next dated catalyst in Alpha-1 Antitrypsin Deficiency is BEAM on 8 Sep 2026. 4 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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