Recent Updates
Recently added Catalysts

Cystic Fibrosis: 4 biotech companies with clinical-stage programmes

Rare Genetic & MetabolicOrphan cornerData as of 2026-09-04

Competitors4in the clinic
Median market cap$605Muniverse median $1.20B
Allocated capital$3.1B$12.5B competing
Phase 3 or filed03 mid, 1 early
Pure plays2this indication only
Capital rank66/299crowding rank 51

Cystic Fibrosis sits in the orphan corner of the map: 4 developers, none of them large, with a median market cap of $605M against a $1.20B universe median.

A thin field of small caps usually means a narrow addressable population, so the whole field can be repriced by one readout or one financing.

KRYS alone accounts for 88% of the $12.5B of market capitalisation competing here, so this is a leader plus a tail rather than a level race.

$3.1B of allocated capital sits in Cystic Fibrosis, 66th of 299 indications, and 2 of the 4 companies are pure plays that trade on this indication alone.

Companies developing drugs for Cystic Fibrosis 4

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
FDMT4D Molecular Therapeutics4D-710Phase 21$768MNo4 30 Sep 2026
ARCTArcturus Therapeutics HoldingsARCT-032Phase 21$442MYes1 30 Sep 2026
SIONSionna TherapeuticsSION-719Phase 24$305MYes1No dated event
KRYSKrystal BiotechKB407Phase 11$11.0BNo5 31 Dec 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile. KRYS holds 88% of the $12.5B competing in this indication.

Why Cystic Fibrosis is a orphan corner

A thin field of small caps. Few competitors, none of them large.

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. Cystic Fibrosis has 4 competitors and a median of $605M.

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

Facioscapulohumeral Muscular Dystrophy2 companies · $3.5B · median $3.5BConcentrated betDystrophic Epidermolysis Bullosa1 company · $2.2B · median $11.0BUncontestedPIK3CA-related Overgrowth Spectrum1 company · $4.2B · median $4.2BUncontestedSickle Cell Disease6 companies · $4.8B · median $2.5BGold rushAcquired Angioedema1 company · $1.2B · median $2.3BUncontestedMECP2 Duplication Syndrome1 company · $1.1B · median $9.7BUncontestedVon Willebrand Disease1 company · $1.0B · median $2.1BUncontestedCongenital FVII Deficiency1 company · $1.0B · median $2.1BUncontested

Cystic Fibrosis pipeline questions

Which biotech companies are developing drugs for Cystic Fibrosis?

4D Molecular Therapeutics (FDMT, 4D-710, Phase 2); Arcturus Therapeutics Holdings (ARCT, ARCT-032, Phase 2); Sionna Therapeutics (SION, SION-719, Phase 2); Krystal Biotech (KRYS, KB407, Phase 1). Those are the 4 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Cystic Fibrosis.

How advanced is the Cystic Fibrosis pipeline?

Of the 4 clinical-stage programmes in Cystic Fibrosis, 0 are in Phase 3 or filed, 3 are in mid-stage development and 1 is in early-stage development.

Which company leads the Cystic Fibrosis field?

KRYS is the largest developer in Cystic Fibrosis, holding 88% of the $12.5B of market capitalisation competing in the indication. The median competitor is worth $605M.

When is the next Cystic Fibrosis catalyst?

The next dated catalyst in Cystic Fibrosis is FDMT on 30 Sep 2026. 3 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.

Back to the disease map