Drugs / AUTOLOGOUS CD34+ ENRICHED CELL FRACTION THAT CONTAINS CD34+ CELLS TRANSDUCED WITH RETROVIRAL VECTOR THAT ENCODES FOR THE HUMAN ADA CDNA SEQUENCE
AUTOLOGOUS CD34+ ENRICHED CELL FRACTION THAT CONTAINS CD34+ CELLS TRANSDUCED WITH RETROVIRAL VECTOR THAT ENCODES FOR THE HUMAN ADA CDNA SEQUENCE
targets ADA via restoration
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Strimvelis | — | 2016-05-26 | europa.eu ↗ |
Trials 7 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT04179643 | Nov 2019 → Aug 2024 overdue | cardiac rhythm disease, diastolic heart failure, ischemic disease, systolic heart failure | AskBio Inc | Active not recruiting | No outcome recorded |
| Phase 1 | NCT01156584 | Jul 2010 → Aug 2016 | anaplastic astrocytoma, anaplastic oligoastrocytoma, anaplastic oligodendroglioma, glioblastoma | Tocagen Inc. | Completed | No outcome recorded |
| Phase 1 | NCT00002824 | Feb 1996 → ? | central nervous system neoplasm | Abramson Cancer Center at Penn Medicine | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04145037 | May 2019 → Aug 2023 | Gaucher disease type I | AVROBIO | Terminated | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT05598333 | Oct 2023 → Oct 2026 expected | congestive heart failure | AskBio Inc | Active not recruiting | No outcome recorded |
| Phase 2 | NCT00598481 | Oct 2002 → Jul 2011 | immunodeficiency disease | Fondazione Telethon | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT05815004 | Oct 2023 → Dec 2027 | Gaucher disease type III | AVROBIO | Withdrawn | No outcome recorded |
Also used as a comparator or background therapy in 1 trial
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 4 | NCT03311074 Background | Jun → Jul 2020 | immune system disorder | Fondazione Telethon | Withdrawn | No outcome recorded |
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AUTOLOGOUS CD34+ ENRICHED CELL FRACTION THAT CONTAINS CD34+ CELLS TRANSDUCED WITH RETROVIRAL VECTOR THAT ENCODES FOR THE HUMAN ADA CDNA SEQUENCE | ChEMBL registry synonym — accepted as the source's own label CHEMBL5315093 ↗ |
| Known as | AB-1002 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05598333 ↗ |
| Known as | AVR-RD-02 | “AVR-RD-02 Drug product: active substance is autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV)...” NCT05815004 ↗ |
| Known as | gene therapy | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00002824 ↗ |
| Known as | GSK2696273 | “GSK2696273 (autologous CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] enzyme) is a gamma-retroviral ex vivo gene therapy” PMID 28319446 ↗ Mar 2017 |
| Known as | Strimvelis | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03311074 ↗ |
| Known as | Toca 511 vector | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01156584 ↗ |
| Action | Restore | “partial to full correction of ADA expression” PMID 19652199 ↗ Aug 2009 |
| Modality | Gene therapy (AAV / viral vector) | “a chimeric cardiotropic adeno-associated viral vector” PMID 41120766 ↗ Oct 2025 |
| Route | Other | “stereotactically injected H5.010RSVTK” NCT00002824 ↗ |
| Target | ADA | “encodes for the human adenosine deaminase [ADA] enzyme” PMID 28319446 ↗ Mar 2017 |