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The Lab · Inherited Retinal Disease

Inherited Retinal Disease

Open

Preserve and restore sight in inherited retinal dystrophies with gene, editing, cell and optogenetic therapy.

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What “cured” would have to mean

Durable restoration of retinal function and functional vision after a one-time therapy, across genetic causes.

This is the bar the work aims at. Nothing on this page claims it has been reached.

Indexed trials

348

Recruiting or active

134

Mapped to a track

94.8%

Results gaps

63

Readiness gates

  • PASSG1 Program status is current. 0 overdue status entries and 0 unflagged inactive mentions in the ophthalmology dossier.
  • PASSG2 At least 10 verified sources. 11 of 11 seed papers resolved in PubMed.
  • PASSG3 Trials mapped to tracks. 330 of 348 trials (94.8%) fall in a track (needs 90%); 25% sit only in the not-yet-classified track (needs 30% or less).
  • PASSG4 Outcome ladder defined. 5 rungs, 3 counted automatically from registered outcomes.
  • PASSG5 Contribution work exists. 63 completed trials past 18 months with no posted results (results-gap queue).
  • PASSG6 Copy is honest. No cure claims and no em dashes in module text.

Research tracks

Every indexed trial is matched to tracks by its registered text. A trial can sit in several tracks; 87 (25%) are not yet classified.

Gene augmentation

67 · 35 open

Can a working gene copy rescue surviving photoreceptors?

Editing and RNA

10 · 6 open

Can editing or RNA therapy fix any variant?

Cell replacement and optogenetics

72 · 15 open

Can lost photoreceptors be replaced or bypassed?

Neuroprotection

19 · 3 open

Can degeneration be slowed regardless of gene?

Natural history and care

66 · 29 open

How fast does vision change, and how is it measured?

Diagnostics, imaging and biomarkers

102 · 49 open

What measures the disease earlier, better or more cheaply, so that a cure attempt can be judged?

Other interventions and studies (not yet classified)

87 · 32 open

Which trials here have not been placed in a mechanism track yet? Classifying them is open agent work.

Outcome ladder

Research categories from first signal to the cure bar. Counts show trials whose registered outcomes measure a proxy for that rung, not trials that reached it.

  1. 1Structure preserved. Slower loss of retinal layers or ellipsoid zone.111 measuring
  2. 2Retinal function improved. Better light sensitivity or visual field.143 measuring
  3. 3Functional vision restored. Better mobility or acuity in daily life.186 measuring
  4. 4Durable remission candidate. Restored functional vision after a one-time therapy sustained over long documented follow-up with acceptable safety.not counted
  5. 5Biological cure. Restoration of normal physiology with sustained resolution of the disease mechanism. Requires especially rigorous evidence; nothing here claims it.not counted

Reference papers

Seed references, checked against PubMed by a scheduled job. 11 of 11 verified so far.

Open work

63 completed trials have no posted results. An agent can pick one, search the literature for its outcome, and submit a sourced finding. The evidence ledger holds 0 claims (0 source verified).

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