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Disease
Severe Combined Immunodeficiency
Late-stage therapeutic developmentEmerging research
1
Publications
21
Clinical trials
2021
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Research2021-05-11The New England journal of medicine
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Low
Key developments
- 3 clinical trials expected to report results, the earliest in Q1 2028.
Upcoming Milestones
- Q1 2028Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
- Q4 2032Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
- Q2 2038Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells
Major developments
Upcoming trial readoutHigh impact
Results expected Q4 20322026-07-08
Upcoming trial readoutImportant
Results expected Q2 20382026-02-13
Clinical MilestonesViewHide
Results expected
- 2026-07-08Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyResults expected Q4 2032
- 2026-02-13Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic CellsResults expected Q2 2038
- 2025-12-11Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningResults expected Q1 2028
Recently completed
- 2026-08-01Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted BusulfanPrimary completion
Activity timeline4
- 2026-08-01ClinicalPhase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted BusulfanPrimary completion
- 2026-07-08ClinicalLentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined ImmunodeficiencyResults expected Q4 2032
- 2026-02-13ClinicalPhase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic CellsResults expected Q2 2038
- 2025-12-11ClinicalPhase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningResults expected Q1 2028
Clinical trials
The current development programme across all trial phases.
Clinical programme
21
6
1
6
Recruiting
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes2
- Genetic Vectors1
- Hematopoietic Stem Cell Transplantation1
Leading journals1
- The New England journal of medicine1
Leading researchers8
- Adams S1
- Arduini S1
- Barman P1
- Booth C1
- Buckland KF1
- Buckley RH1
- Campo Fernandez B1
- Candotti F1
Affiliations (unnormalised)1
- and Pathology and Laboratory Medicine (G.M.C.) and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research (D.B.K.1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
Group of rare congenital disorders characterized by impairment of both humoral and cell-mediated immunity, leukopenia, and low or absent antibody levels. It is inherited as an X-linked or autosomal recessive defect. Mutations occurring in many different genes cause human Severe Combined Immunodeficiency (SCID).
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.