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Advancing Treatment of Hemoglobinopathies: Precision Gene-Editing and Base Editing to Restore Fetal Hemoglobin Production
Advancing Treatment of Hemoglobinopathies: Precision Gene-Editing and Base Editing to Restore Fetal Hemoglobin Production
Background
Hemoglobinopathies, such as β-thalassemias and sickle cell anemia, are genetic disorders with significant impact on global health, affecting millions with severe anemia due to abnormal or deficient β-globin....
Published: 3/24/2026
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Updated: 3/24/2026
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Inventor(s):
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Disease Area > Hematology
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A base editing approach for Shwachman-Diamond-Syndrome
A base editing approach for Shwachman-Diamond-Syndrome
Background
Shwachman-Diamond-Syndrome (SDS), is life threatening genetic condition caused in 90% of cases by a mutation in the SBDS-gene, which leads to defective splicing and loss of protein expression. Patients may develop many symptoms such as frequent infections due to low white blood cell...
Published: 3/26/2026
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Updated: 12/8/2025
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Inventor(s): Christian Brendel
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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A specific chemical and transcription factor combination specifies and expands hematopoietic stem and progenitor cells in zebrafish embryonic cell culture.
Method for Inducing Hematopoietic Stem Cell Specificity
Background
Hematopoietic stem cells (HSCs), found primarily in bone marrow, are crucial for blood cell formation and immune system function. While transplantation of allogeneic and autologous HSCs is common for treating various life-threatening disorders, the procedure carries high risks, primarily...
Published: 3/26/2026
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Updated: 11/4/2025
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Inventor(s): Leonard Zon, Joseph Mandelbaum, Anne Robertson
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Small Molecule
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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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Advanced CRISPR/Cas Technology for Targeted Fetal Hemoglobin Reinduction in Hemoglobinopathy Treatment
Advanced CRISPR/Cas Technology for Targeted Fetal Hemoglobin Reinduction in Hemoglobinopathy Treatment
Background
Hemoglobinopathies, such as β-thalassemias and sickle cell anemia, represent a significant unmet medical need affecting millions globally. These genetic disorders are characterized by defective or deficient production of normal β-globin,...
Published: 3/26/2026
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Updated: 10/9/2025
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Inventor(s): Daniel Bauer
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Therapeutics > Gene Therapy
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Hematopoietic Stem Cell Transplantation: Enhanced Engraftment without Chemotherapy for Genetic and Acquired Disorders
Hematopoietic Stem Cell Transplantation: Enhanced Engraftment without Chemotherapy for Genetic and Acquired Disorders
Background
There is a crucial unmet medical need for improved methods to treat inherited and acquired diseases via hematopoietic stem cell transplantation. Currently, patients must undergo high-dose alkylating chemotherapy as part...
Published: 3/26/2026
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Updated: 10/9/2025
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Inventor(s): Daniel Bauer, Vivien Schoonenberg, Sabine Studer, Sebastien Levesque
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Cell Therapy, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Disease Area > Hematology
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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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Targeting cytokine secretion to ameliorate fibrosis in myeloproliferative neoplasms
Targeting cytokine secretion to ameliorate fibrosis in myeloproliferative neoplasms
Background
Myelofibrosis is a bone marrow cancer characterized by disruptions in blood cell production. This leads to several symptoms such as anemia and an increased risk of infection and bleeding. Primary and secondary myelofibrosis, a subset of myeloproliferative...
Published: 3/30/2026
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Updated: 3/10/2025
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Inventor(s): Joseph Italiano, Isabelle Becker
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology, Technology Classifications > Invention Type > Novel Targets
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Production of T-cells using engineered soluble DLL4 ligands
Production of T cells using engineered soluble DLL4 ligands
Background
Chimeric Antigen Receptor T-cell (CAR T-cell) therapies are a therapeutic tool for the treatment of several types of cancers, most notably lymphomas and leukemia. Healthy T cells are taken from the patient’s blood, cultured in the laboratory to induce differentiation, and...
Published: 3/26/2026
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Updated: 11/20/2024
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Inventor(s): Rubul Mout, Ran Jing, George Daley, Stephen Blacklow, David Baker, Emily Egan
Keywords(s):
Category(s): Technology Classifications > Invention Type > Therapeutics > Cell Therapy, Technology Classifications > Disease Area > Immunology, Technology Classifications > Disease Area > Hematology
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Genetic regulators of fetal hemoglobin
Genetic Regulators of fetal hemoglobin
Background
During human development, the major hemoglobin in red blood cells changes from fetal hemoglobin (HbF, α₂γ₂) to adult hemoglobin (HbA, α₂β₂) shortly after birth. This change, known as the fetal-to-adult hemoglobin switch, involves a transcriptional shift from the γ-globin...
Published: 3/26/2026
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Updated: 11/20/2024
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Inventor(s): Vijay Sankaran, Liam Cato, Rick Li, Yi-Chin Lu, Chun-Jie Guo
Keywords(s):
Category(s): Technology Classifications, Technology Classifications > Disease Area > Hematology, Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Novel Targets
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