Disease

Cone rod dystrophy

Active therapeutic pipeline

Clinical trials

Associated genes

Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low

Key developments

1 clinical trial expected to report results, the earliest in Q3 2030.

Upcoming Milestones

Q3 2030ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy

Major developments

Upcoming trial readoutImportant

ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy

Results expected Q3 20302025-12-02

Clinical Milestones1View

Results expected 1

2025-12-02ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related RetinopathyResults expected Q3 2030

Activity timeline1

2025-12-02ClinicalACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related RetinopathyResults expected Q3 2030

Clinical trials

1 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Recruiting

ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy

Phase 1/2 · Recruiting · Ascidian Therapeutics, Inc

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

POC1B0.77 GUCA1A0.76 RPGR0.75 DRAM20.75 CEP780.73 PDE6C0.72 TTLL50.72 ADAM90.72 RPGRIP10.71 CRX0.71 RAB280.71 ABCA40.71

Disease biology

2 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Voltage-dependent L-type calcium channel subunit alpha-1Fvia CACNA1F0.69 Potassium voltage-gated channel subfamily V member 2via KCNV20.45

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.