Disease · MONDO_0007064
severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency. Diseases, record MONDO_0007064. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0007064
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
- severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency
- Open Targets
- Other names
ADA deficiency, ADA-SCID, SCID due to ADA deficiency, SCID due to ADA deficiency, early-onset, SCID due to adenosine deaminase deficiency, Severe combined immunodeficiency due to adenosine deaminase deficiency, adenosine deaminase deficiency, adenosine deaminase deficiency, partial, Autosomal recessive, Somatic mosaicism, adenosine deaminase deficient severe combined immunodeficiency, severe combined immunodeficiency due to ADA deficiency, Autosomal recessive, Somatic mosaicism
- Open Targets
Drugs2
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 |
|---|---|---|---|---|---|---|---|---|
| SIMOLADAGENE AUTOTEMCEL | CHEMBL4298201 | Gene | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| PEGADEMASE BOVINE | CHEMBL1201514 | Enzyme | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets0
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.
None in this release.
Trials2
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 |
|---|---|---|---|---|---|---|---|---|
| NCT01380990 | Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Phase 1/2 | 36 | 2021-09-16 | Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID) | PEGADEMASE BOVINE | Its record names the disease | ClinicalTrials.gov |
| NCT04140539 | A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 | Phase 2/3 | 0 | Severe Combined Immunodeficiency Due to ADA Deficiency | SIMOLADAGENE AUTOTEMCEL | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials2
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 |
|---|---|---|---|---|---|
| OTL-101* On-Study and CUP Subjects vs OTL-101* On-Study Subjects: deaths from any cause | 0 of 20 | 0 of 10 | 30 | NCT01380990 | Trial comparisons |
| OTL-101* On-Study and CUP Subjects vs OTL-101* On-Study Subjects: serious adverse events | 11 of 20 | 7 of 10 | 30 | NCT01380990 | Trial comparisons |
Papers from its trials1
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 |
|---|---|---|
| 33974366 | DERIVED | 1 |
Source PubMed
Data dates
The date of each source this release read.
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_0007064
GET https://conductscience.org/api/diseases/v1/MONDO_0007064/drugs