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Leber Congenital Amaurosis: two biotech companies racing to a treatment

OphthalmologyOrphan cornerData as of 2026-09-04

Competitors2in the clinic
Median market cap$300Muniverse median $1.20B
Allocated capital$600M$600M competing
Phase 3 or filed01 mid, 1 early
Pure plays2this indication only
Capital rank194/299crowding rank 95

Leber Congenital Amaurosis is a two-horse race: ProQR Therapeutics (PRQR, $220M) against Opus Genetics (IRD, $380M).

A duopoly prices differently from an empty field. Each company is the other's comparable, so a readout from either side repositions both, and $600M of allocated capital sits on the pair.

IRD carries 63% of the $600M competing here, so the pair is far from evenly matched.

$600M of allocated capital sits in Leber Congenital Amaurosis, 194th of 299 indications, and 2 of the 2 companies are pure plays that trade on this indication alone.

Companies developing drugs for Leber Congenital Amaurosis 2

TickerCompanyLead assetPhaseTrialsMarket capPure playIndicationsNext catalyst
PRQRProQR TherapeuticssepofarsenPhase 21$220MYes1 31 Dec 2026
IRDOpus GeneticsOPGx-RDH12Phase 11$380MYes1 9 Sep 2026

Sorted by phase, then by market capitalisation. Ticker links open the company profile. IRD holds 63% of the $600M competing in this indication.

Why Leber Congenital Amaurosis 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. Leber Congenital Amaurosis has 2 competitors and a median of $300M.

Ophthalmology

Ophthalmology holds $17.0B of the map's capital, 2.5% of the total, spread across 9 indications and 13 companies. 6 of those indications have more than one company competing.

See every Ophthalmology indication on the map

Related indications in Ophthalmology

Macular Edema1 company · $758M · median $2.3BUncontestedX-Linked Retinitis Pigmentosa1 company · $192M · median $768MUncontestedHereditary Transthyretin Amyloidosis1 company · $1.1B · median $9.7BUncontestedDiabetic Macular Edema4 companies · $1.3B · median $613MOrphan cornerDry Eye Disease3 companies · $2.0B · median $706MOrphan cornerWet Age-related Macular Degeneration4 companies · $3.5B · median $1.5BConcentrated betStargardt Disease2 companies · $3.6B · median $3.7BConcentrated betGeographic Atrophy3 companies · $4.0B · median $918MOrphan corner

Leber Congenital Amaurosis pipeline questions

Which biotech companies are developing drugs for Leber Congenital Amaurosis?

ProQR Therapeutics (PRQR, sepofarsen, Phase 2); Opus Genetics (IRD, OPGx-RDH12, Phase 1). Those are the 2 US-listed developers in the $100M to $20B market cap band with a clinical-stage programme in Leber Congenital Amaurosis.

How advanced is the Leber Congenital Amaurosis pipeline?

Of the 2 clinical-stage programmes in Leber Congenital Amaurosis, 0 are in Phase 3 or filed, 1 is in mid-stage development and 1 is in early-stage development.

Which company leads the Leber Congenital Amaurosis field?

IRD is the largest developer in Leber Congenital Amaurosis, holding 63% of the $600M of market capitalisation competing in the indication. The median competitor is worth $300M.

When is the next Leber Congenital Amaurosis catalyst?

The next dated catalyst in Leber Congenital Amaurosis is IRD on 9 Sep 2026. 2 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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