AUTOLOGOUS CD34+ ENRICHED CELL FRACTION THAT CONTAINS CD34+ CELLS TRANSDUCED WITH RETROVIRAL VECTOR THAT ENCODES FOR THE HUMAN ADA CDNA SEQUENCE
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Also known as Strimvelis
Summary
Autologous Cd34+ Enriched Cell Fraction That Contains Cd34+ Cells Transduced With Retroviral Vector That Encodes For The Human Ada Cdna Sequence (CHEMBL5315093) is an approved unknown; indicated across 1 condition including severe combined immunodeficiency.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Unknown
- Indications: 1 condition
- Clinical trials: 4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL5315093 |
| Name | AUTOLOGOUS CD34+ ENRICHED CELL FRACTION THAT CONTAINS CD34+ CELLS TRANSDUCED WITH RETROVIRAL VECTOR THAT ENCODES FOR THE HUMAN ADA CDNA SEQUENCE |
| Type | Unknown |
| Max phase | 4 |
Also known as: Strimvelis
Targets
Targets
No target linkage available.
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
1 indication (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| severe combined immunodeficiency | 4 | MONDO:0015974 | MONDO:0015974 |
Clinical trials
Total trials: 4.
Phase distribution
| Phase | Trials |
|---|---|
| Not specified | 3 |
| PHASE4 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT03311074 | PHASE4 | WITHDRAWN | Retroviral Insertion Site Methodology Study |
| NCT03478670 | Not specified | ENROLLING_BY_INVITATION | Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) |
| NCT03232203 | Not specified | COMPLETED | Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs) |
| NCT04959890 | Not specified | UNKNOWN | Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).
Related Atlas pages
- Diseases: severe combined immunodeficiency