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Advancing Hemoglobinopathy Care: Innovative BCL11A Inhibition for Sustained Fetal Hemoglobin Production
Advancing Hemoglobinopathy Care: Innovative BCL11A Inhibition for Sustained Fetal Hemoglobin Production
Background
Hemoglobinopathies, including sickle cell anemia and β-thalassemias, are genetic disorders caused by mutations that impair the production or structure of of β-globin chains in hemoglobin. These conditions result in patients...
Published: 3/25/2026
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Updated: 12/5/2025
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Inventor(s): Daniel Bauer, Yuxuan Wu, Jing Zeng, Scot Wolfe, Mehmet Bolukbasi, Benjamin Roscoe, Pengpeng Liu, Luk.Kevin
Keywords(s): Sickle Cell Anemia
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Enhanced BCL11A RNP / CRISPR delivery & editing using a 3xNLS-Cas9
Advancing Gene Therapy for Hemoglobinopathies: Enhanced CRISPR-Cas9 with Multi-NLS Integration
Background
Sickle cell disease (SCD) and β-thalassemia are severe β-globin disorders affecting millions globally, characterized by defective adult hemoglobin that fails to transport oxygen effectively, leading to significant morbidity. Existing...
Published: 3/25/2026
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Updated: 10/13/2025
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Inventor(s): Daniel Bauer, Pengpeng Liu, Scot Wolfe, Yuxuan Wu, Jing Zeng
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Expanding HSCs by blocking ferroptosis
Expanding HSCs by blocking ferroptosis
Background
Hematopoietic stem cell (HSC) transplant is a common life-saving treatment for several hematological and immunological disorders, including leukemia, bone marrow failure syndromes, and immunodeficiencies. To date, over 1.5 million HSC transplants have been performed, making it a widespread therapeutic...
Published: 3/26/2026
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Updated: 10/9/2025
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Inventor(s): Vijay Sankaran, Lucrezia Della Volpe
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Therapeutic twin prime editing of hematopoietic stem cells for TINF2 dyskeratosis congenita
Therapeutic twin prime editing of hematopoietic stem cells for TINF2 dyskeratosis congenita
Background
Dyskeratosis congenita (DC) is a rare, genetic form of bone marrow (BM) failure. It is estimated that one in one million people has this condition. About 50% of the people who have DC have changes in the genes that are important in telomere maintenance,...
Published: 3/26/2026
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Updated: 11/20/2024
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Inventor(s): Daniel Bauer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Editing
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Improving gene editing by modulating nucleotide metabolism
Improving gene editing by modulating nucleotide metabolism
Background
Currently, most treatments for genetic blood-related disorders (both non-cancerous and cancerous) aim to alleviate symptoms, manage complications, and slow disease progression, rather than completely correcting the underlying genetic defect. In some cases, patients may qualify...
Published: 3/26/2026
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Updated: 11/20/2024
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Inventor(s): Daniel Bauer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Gene Editing, Technology Classifications > Disease Area > Rare Diseases
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