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HBB

—Hemoglobin Subunit Beta

Chromosome: 11

Location: 11:5225464-5229395

OMIM: 141900

Ensembl: ENSG00000244734

Official·OMIM:141900 Official·ENSG00000244734

Encodes the beta subunit of hemoglobin. Mutations cause sickle cell disease and beta-thalassemia.

Treatment Roadmap — HBB

FDA Approved

Preclinical

Phase 1

2 trial

Phase 2

4 trial

Phase 3

1 trial

Approved

Casgevy (exagamglogene autotemcel) — FDA (1/16/2024)

Clinical trials have approximately 10% success rate. This roadmap shows the current status, not guaranteed outcomes.

Associated Diseases

Sickle Cell Disease

Autosomal RecessivePrevalence: ~7.7Mhemoglobin

Official·OMIM:603903 Official·ORPHA232

Beta-Thalassemia

Autosomal RecessivePrevalence: ~5Mhemoglobin

Official·

HBB

Treatment Roadmap — HBB

Associated Diseases

Summary

Approved Therapies