Disease · MONDO_0010557

choroideremia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. choroideremia. Diseases, record MONDO_0010557. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010557

On this page

Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: choroideremia; Open Targets
Open Targets ID: MONDO_0010557; Open Targets
Other names: CHM, Tapetochoroidal dystrophy, progressive choroidal atrophy; Open Targets
Disease Ontology: DOID:9821; Disease Ontology
MeSH: D015794; MeSH
NCI Thesaurus: C34469; NCI Thesaurus
Orphanet: ORPHA:180; Orphanet
Highest stage of its drugs: Phase 2, for 2 of its 4 drugs, from ClinicalTrials.gov; Open Targets

Drugs4

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
CILIARY NEUROTROPHIC FACTOR	CHEMBL4298063		Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
TIMREPIGENE EMPARVOVEC	CHEMBL4594315	Gene	Phase 2	ClinicalTrials.gov	2	1	1	Drugs and targets
KIO-301	CHEMBL4796560	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
SIMVASTATIN	CHEMBL1064	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets1

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes
HMGCR	3-hydroxy-3-methylglutaryl-coenzyme A reductase	1	HMG-CoA reductase inhibitor	Phase 1/2	https://conductscience.org/library/genes/3156

Source Drugs and targets

Trials5

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00447980	A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa	Phase 2	73	2025-05-01	Retinitis Pigmentosa	CILIARY NEUROTROPHIC FACTOR	Its record names the disease	ClinicalTrials.gov
NCT00447993	A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa	Phase 2	67	2024-08-13	Retinitis Pigmentosa	CILIARY NEUROTROPHIC FACTOR	Its record names the disease	ClinicalTrials.gov
NCT03507686	A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111	Phase 2	66	2023-09-18	Choroideremia	TIMREPIGENE EMPARVOVEC	Its record names the disease	ClinicalTrials.gov
NCT05282953	A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)	Phase 1/2	32		Retinitis Pigmentosa, Choroideremia, Non-Syndromic Rod-Dominant Inherited Retinal Diseases	KIO-301	Its record names the disease	ClinicalTrials.gov
NCT01654562	The Short-term Effects of Simvastatin on the Vision of Males Affected by Choroideremia	Phase 1/2	2		Choroideremia	SIMVASTATIN	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials7

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Treatment Period 1: BIIB111 vs Treatment Period 2: BIIB111: Number of Participants With Anti-drug Antibodies Post-treatment at Month 12	0 of 60	0 of 56	116	NCT03507686	Trial comparisons
Treatment Period 1: BIIB111 vs Treatment Period 2: BIIB111: deaths from any cause	0 of 60	1 of 56	116	NCT03507686	Trial comparisons
Treatment Period 1: BIIB111 vs Treatment Period 2: BIIB111: serious adverse events	12 of 60	8 of 56	116	NCT03507686	Trial comparisons
Low Dose vs High Dose: deaths from any cause	0 of 20	0 of 48	68	NCT00447980	Trial comparisons
Low Dose vs High Dose: serious adverse events	1 of 20	4 of 48	68	NCT00447980	Trial comparisons
Low Dose vs High Dose: deaths from any cause	0 of 22	0 of 43	65	NCT00447993	Trial comparisons
Low Dose vs High Dose: serious adverse events	1 of 22	0 of 22	44	NCT00447993	Trial comparisons

Papers from its trials6

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
41981307	DERIVED	1	PubMed
38970425	DERIVED	1	PubMed
30194931	DERIVED	1	PubMed
23668681	DERIVED	2	PubMed
23049090	DERIVED	2	PubMed
21087953	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0010557

GET https://conductscience.org/api/diseases/v1/MONDO_0010557/drugs

API reference For AI assistants