NORAD RNA Treatment for Beta-Thalassemia, Sickle Cell Disease
Summary
USPTO published patent application US20260109984A1 for therapeutic methods, systems, and compositions treating hemoglobinopathies including beta-thalassemia and sickle cell disease by increasing expression of noncoding RNA activated by DNA damage (NORAD). The application was filed on 2025-10-28 under application number 19371206, with inventors Merlin Nithya Gnanapragasam and Mahesh Ramamoorthy. Overexpression of NORAD long non-coding RNA is shown to increase fetal hemoglobin expression, providing a therapeutic approach for these genetic blood disorders.
“Therapeutic methods, systems, and compositions for treating a patient suffering from hemoglobinopathy include treating the patient to increase expression of noncoding RNA activated by DNA damage (NORAD).”
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GovPing monitors USPTO Patent Applications - Biotech (C12N) for new healthcare & life sciences regulatory changes. Every update since tracking began is archived, classified, and available as free RSS or email alerts — 263 changes logged to date.
What changed
USPTO published patent application US20260109984A1 disclosing methods, systems, and compositions for treating hemoglobinopathies such as beta-thalassemia and sickle cell disease. The invention involves increasing expression of noncoding RNA activated by DNA damage (NORAD), where overexpression of NORAD long non-coding RNA leads to increased fetal hemoglobin expression.
Biotechnology and pharmaceutical companies developing gene therapies or treatments for genetic blood disorders should review this patent application for potential overlap with their own R&D programs or licensing considerations. The therapeutic approach using NORAD RNA modulation may represent a novel pathway for treating hemoglobinopathies.
Archived snapshot
Apr 23, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
METHODS, SYSTEMS, AND COMPOSITIONS FOR TREATMENT OF HEMOGLOBINOPATHY (E.G., BETA-THALASSEMIA AND SICKLE CELL DISEASE) BY INCREASING EXPRESSION OF NONCODING RNA ACTIVATED BY DNA DAMAGE
Application US20260109984A1 Kind: A1 Apr 23, 2026
Inventors
Merlin Nithya Gnanapragasam, Mahesh Ramamoorthy
Abstract
Therapeutic methods, systems, and compositions for treating a patient suffering from hemoglobinopathy include treating the patient to increase expression of noncoding RNA activated by DNA damage (NORAD). Overexpression of NORAD long non-coding RNA leads to an increase in fetal hemoglobin expression. The hemoglobinopathy may be β-thalassemia or sickle cell disease.
CPC Classifications
C12N 15/1136 A61K 9/1272 A61K 9/5123 A61P 7/06 C12N 15/88 B82Y 5/00 C12N 2310/11 C12N 2310/14
Filing Date
2025-10-28
Application No.
19371206
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