Disease · MONDO_0006541

epidermolysis bullosa

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. epidermolysis bullosa. Diseases, record MONDO_0006541. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0006541

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: epidermolysis bullosa; Open Targets
Open Targets ID: MONDO_0006541; Open Targets
Other names: acantholysis bullosa; Open Targets
Disease Ontology: DOID:2730; Disease Ontology
MeSH: D004820; MeSH
NCI Thesaurus: C67383; NCI Thesaurus
Highest stage of its drugs: Approval, for 1 of its 20 drugs, from TTD; Open Targets

Drugs20

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
BIRCH TRITERPENES	CHEMBL4650246	Small molecule	Approval	TTD	2	0	0	Drugs and targets
ALLANTOIN	CHEMBL593429	Small molecule	Phase 3	ClinicalTrials.gov	5	5	5	Drugs and targets
BEREMAGENE GEPERPAVEC	CHEMBL4594328	Gene	Phase 3	TTD	1	0	0	Drugs and targets
CANNABIDIOL	CHEMBL190461	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
BUSULFAN	CHEMBL820	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
NELITOLIMOD	CHEMBL6068457	Oligonucleotide	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
CANNABINOL	CHEMBL74415	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DIACEREIN	CHEMBL41286	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 20

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Targets16

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	Source
CNR1	Cannabinoid receptor 1	1	Cannabinoid CB1 receptor negative allosteric modulator	Phase 2/3	https://conductscience.org/library/genes/1268	Drugs and targets
COL7A1	Collagen alpha-1(VII) chain	1	Collagen alpha-1(VII) chain exogenous gene	Phase 3	https://conductscience.org/library/genes/1294	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	1	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
IMPDH1	Inosine-5'-monophosphate dehydrogenase (IMPDH)	1	Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor	Phase 2	https://conductscience.org/library/genes/3614	Drugs and targets
IMPDH2	Inosine-5'-monophosphate dehydrogenase (IMPDH)	1	Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor	Phase 2	https://conductscience.org/library/genes/3615	Drugs and targets
SCN10A	Sodium channel alpha subunit	1	Sodium channel alpha subunit blocker	Unknown	https://conductscience.org/library/genes/6336	Drugs and targets
SCN11A	Sodium channel alpha subunit	1	Sodium channel alpha subunit blocker	Unknown	https://conductscience.org/library/genes/11280	Drugs and targets
SCN1A	Sodium channel alpha subunit	1	Sodium channel alpha subunit blocker	Unknown	https://conductscience.org/library/genes/6323	Drugs and targets
SCN2A	Sodium channel alpha subunit	1	Sodium channel alpha subunit blocker	Unknown	https://conductscience.org/library/genes/6326	Drugs and targets
SCN3A	Sodium channel alpha subunit	1	Sodium channel alpha subunit blocker	Unknown	https://conductscience.org/library/genes/6328	Drugs and targets

1 to 10 of 16

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Trials16

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
NCT02384460	ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa	Phase 3	169	2018-10-16	Epidermolysis Bullosa	ALLANTOIN	Its record names the disease	ClinicalTrials.gov
NCT02014376	Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa	Phase 2	48	2020-01-13	Epidermolysis Bullosa	ALLANTOIN, NELITOLIMOD	Its record names the disease	ClinicalTrials.gov
NCT02090283	Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa	Phase 2	42	2019-11-04	Epidermolysis Bullosa	ALLANTOIN, NELITOLIMOD	Its record names the disease	ClinicalTrials.gov
NCT01033552	Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs	Phase 1/2	32	2024-04-03	Epidermolysis Bullosa	ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE), BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT00311766	A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa	Phase 2	30	2013-06-10	Epidermolysis Bullosa	THYMOSIN	Its record names the disease	ClinicalTrials.gov
NCT02582775	MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs	Phase 2	17	2024-09-19	Epidermolysis Bullosa	BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE, MYCOPHENOLATE MOFETIL, TACROLIMUS ANHYDROUS	Its record names the disease	ClinicalTrials.gov
NCT02654483	Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients	Phase 2	14	2019-07-02	Epidermolysis Bullosa, Pruritus	SERLOPITANT	Its record names the disease	ClinicalTrials.gov
NCT00825565	Study of Alwextin® Cream in Treating Epidermolysis Bullosa	Phase 2	8	2013-01-01	Epidermolysis Bullosa	ALLANTOIN	Its record names the disease	ClinicalTrials.gov
NCT07482813	An Open Label Extension Safety Study to Evaluate SD-101 in Epidermolysis Bullosa	Phase 3	80		Epidermolysis Bullosa (EB)	ALLANTOIN	Its record names the disease	ClinicalTrials.gov
NCT04908215	INM-755 (Cannabinol) Cream for Treatment of Epidermolysis Bullosa	Phase 2	19		Epidermolysis Bullosa Simplex, Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica, Kindler Syndrome	CANNABINOL	Its record names the disease	ClinicalTrials.gov

1 to 10 of 16

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Comparisons in its trials4

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
SD-101-6.0 Cream vs Placebo (SD-101-0.0) Cream: deaths from any cause	0 of 82	1 of 87	169	NCT02384460	Trial comparisons
SD-101-6.0 Cream vs Placebo (SD-101-0.0) Cream: serious adverse events	4 of 82	8 of 87	169	NCT02384460	Trial comparisons
Thymosin Beta 4 vs Placebo: Number of Participants Whose Wounds Have Healed	8 of 22	5 of 8	30	NCT00311766	Trial comparisons
Thymosin Beta 4 vs Placebo: serious adverse events	0 of 22	0 of 8	30	NCT00311766	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
32939751	RESULT	1	PubMed
31541747	RESULT	1	PubMed
28821470	RESULT	1	PubMed
32693833	DERIVED	1	PubMed
32576219	DERIVED	1	PubMed
30843184	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_0006541

GET https://conductscience.org/api/diseases/v1/MONDO_0006541/drugs

API reference For AI assistants