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ADA gene transfer
ADA gene transfer involves the introduction of a healthy copy of the ADA gene into a patient's hematopoietic stem cells to treat ADA-SCID.
ADA gene transfer involves the introduction of a healthy copy of the ADA gene into a patient's hematopoietic stem cells to treat ADA-SCID. Used for Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.
At a glance
| Generic name | ADA gene transfer |
|---|---|
| Sponsor | Donald B. Kohn, M.D. |
| Target | ADA gene |
| Modality | Biologic |
| Therapeutic area | Immunology |
| Phase | Phase 2 |
Mechanism of action
This approach aims to restore the patient's immune system by providing a functional ADA enzyme, which is essential for the breakdown of toxic substances in the body. The healthy ADA gene is introduced into the patient's hematopoietic stem cells, which then produce healthy immune cells that can function properly. This treatment has shown promise in treating patients with ADA-SCID, a rare and severe immunodeficiency disorder.
Approved indications
- Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Common side effects
- Gene therapy-related adverse events
Key clinical trials
- An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
- Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA) (NA)
- Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector (PHASE1, PHASE2)
- Retroviral Insertion Site Methodology Study (PHASE4)
- ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID (PHASE2)
- Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
- Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
- Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ADA gene transfer CI brief — competitive landscape report
- ADA gene transfer updates RSS · CI watch RSS
- Donald B. Kohn, M.D. portfolio CI