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Late-onset Pompe Disease: one US-listed biotech with a clinical-stage programme

Rare Genetic & MetabolicUncontestedData as of 2026-09-04

Competitors1in the clinic
Median market cap$3.6Buniverse median $1.20B
Allocated capital$729M$3.6B competing
Phase 3 or filed00 mid, 1 early
Pure plays0this indication only
Capital rank174/299crowding rank 185

Late-onset Pompe Disease is uncontested. Denali Therapeutics (DNLI) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with DNL952 in phase 1.

There is no public comparable to price it against. DNLI spreads its $3.6B market cap across 5 indications, so $729M of it is allocated here.

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

$729M of allocated capital sits in Late-onset Pompe Disease, ranking it 174th of 299 indications by the money committed to it.

Companies developing drugs for Late-onset Pompe Disease 1

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
DNLIDenali TherapeuticsDNL952Phase 11$3.6BNo5 31 Dec 2026

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

Why Late-onset Pompe Disease 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. Late-onset Pompe Disease has one competitor and a median of $3.6B.

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

Mucopolysaccharidosis Type IIIA1 company · $729M · median $3.6BUncontestedSanfilippo Syndrome (MPS IIIB)2 companies · $654M · median $815MOrphan cornerLimb-Girdle Muscular Dystrophy1 company · $809M · median $2.4BUncontestedErythropoietic Protoporphyria1 company · $609M · median $3.0BUncontestedOsteogenesis Imperfecta Type IV1 company · $488M · median $1.5BUncontestedConstipation Predominant Irritable Bowel Syndrome1 company · $462M · median $924MUncontestedParoxysmal Nocturnal Hemoglobinuria1 company · $461M · median $1.4BUncontestedGuillain-Barré Syndrome1 company · $459M · median $918MUncontested

Late-onset Pompe Disease pipeline questions

Which biotech companies are developing drugs for Late-onset Pompe Disease?

Denali Therapeutics (DNLI, DNL952, Phase 1). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Late-onset Pompe Disease.

How advanced is the Late-onset Pompe Disease pipeline?

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

Is anyone else developing a drug for Late-onset Pompe Disease?

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

When is the next Late-onset Pompe Disease catalyst?

The next dated catalyst in Late-onset Pompe Disease is DNLI 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.

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