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beta-thalassemia major - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

beta-thalassemia major

MONDO:0016486

Also known as:Cooley anaemiaCooley anemiaCooley's Anaemia

Find Clinical TrialsTrials

Overview

Beta-thalassemia (BT) major is a severe early-onset form of BT characterized by severe anemia requiring regular red blood cell transfusions.

Treatments2 FDA approved, incl. 2 gene therapies, newest 2024

Clinical Trials52 registered · 17 recruiting

Diagnosis

Biomarker and diagnostic research for beta-thalassemia major has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

2 FDA-approved treatments are available for beta-thalassemia major, including exagamglogene autotemcel (Casgevy, approved 2024) and betibeglogene autotemcel (Zynteglo, approved 2022). An additional 1 compound holds orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

52 clinical trials registered, 17 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and procedural interventions. Pipeline includes 1 PHASE4, 5 PHASE3, 9 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT07112703](https://clinicaltrials.gov/study/NCT07112703)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 2:52 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:231214

GARD

Genetic and Rare Diseases Info Center

GARD:17162

Questions for Your Care Team

→What are the eligibility criteria and monitoring requirements for Casgevy?
→Are any of the 52 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
Lentiviral vector encoded with a human beta-globin gene plasmid	Lentiviral vector encoded with a human beta-globin gene plasmid	San Rocco Therapeutics, LLC	2006	—	Designated

Research Type	Count	% of Total
Disease patterns and progression	26	33%
Laboratory research	18	23%
Research summaries	13	16%
Clinical study results	9	11%
Testing and diagnosis research	5	6%
Patient case studies	5	6%
New treatment approaches	2	3%
Other research	1	1%

Kisho

beta-thalassemia major

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications

beta-thalassemia major

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Clinical Trials

Recent Publications