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Mucopolysaccharidosis Type II (MPS II): one US-listed biotech with a clinical-stage programme

Rare Genetic & MetabolicUncontestedData as of 2026-09-04

Competitors1in the clinic
Median market cap$607Muniverse median $1.20B
Allocated capital$304M$607M competing
Phase 3 or filed10 mid, 0 early
Pure plays0this indication only
Capital rank235/299crowding rank 238

Mucopolysaccharidosis Type II (MPS II) is uncontested. REGENXBIO (RGNX) is the only US-listed developer in the $100M to $20B band running a clinical programme here, with RGX-121-3102 in phase 3.

There is no public comparable to price it against. RGNX spreads its $607M market cap across 2 indications, so $304M of it is allocated here.

The one programme here is in late-stage development, with 1 dated catalyst in the next 12 months.

$304M of allocated capital sits in Mucopolysaccharidosis Type II (MPS II), ranking it 235th of 299 indications by the money committed to it.

Companies developing drugs for Mucopolysaccharidosis Type II (MPS II) 1

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
RGNXREGENXBIORGX-121-3102Phase 32$607MNo2 30 Sep 2026

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

Why Mucopolysaccharidosis Type II (MPS II) 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. Mucopolysaccharidosis Type II (MPS II) has one competitor and a median of $607M.

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

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Mucopolysaccharidosis Type II (MPS II) pipeline questions

Which biotech companies are developing drugs for Mucopolysaccharidosis Type II (MPS II)?

REGENXBIO (RGNX, RGX-121-3102, Phase 3). Those are the 1 US-listed developer in the $100M to $20B market cap band with a clinical-stage programme in Mucopolysaccharidosis Type II (MPS II).

How advanced is the Mucopolysaccharidosis Type II (MPS II) pipeline?

Of the 1 clinical-stage programme in Mucopolysaccharidosis Type II (MPS II), 1 is in Phase 3 or filed, 0 are in mid-stage development and 0 are in early-stage development.

Is anyone else developing a drug for Mucopolysaccharidosis Type II (MPS II)?

No. RGNX is the only US-listed company in the $100M to $20B market cap band with a clinical-stage programme in Mucopolysaccharidosis Type II (MPS II), which is why the indication is classified as uncontested.

When is the next Mucopolysaccharidosis Type II (MPS II) catalyst?

The next dated catalyst in Mucopolysaccharidosis Type II (MPS II) is RGNX on 30 Sep 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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