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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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Targeting the Super Elongation Complex to Prevent Hematopoietic Stem Cell Expansion and Blood Cancer Risk
Targeting the Super Elongation Complex to Prevent Hematopoietic Stem Cell Expansion and Blood Cancer Risk
Background
It is estimated that over 1.7 million people in the United States are living with or in remission from blood cancers such as leukemias, lymphomas, myeloma, and myeloproliferative neoplasms (MPNs), and approximately 192,070 new cases...
Published: 3/26/2026
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Updated: 12/19/2025
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Inventor(s): Vijay Sankaran, Jiawei Zhao, Liam Cato
Keywords(s): Blood Cancer
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Novel Targets
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Novel Telomere Regulation Technology
Novel Telomere Regulation Technology
Background
Telomeres, protective caps at the ends of chromosomes, play a crucial role in maintaining cellular health. Their length is a key determinant of a cell's ability to replicate. Shortened telomeres, due to genetic mutations or acquired environmental insults, lead to telomerase dysfunction. This has...
Published: 3/23/2026
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Updated: 12/5/2025
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Inventor(s): Suneet Agarwal, Baris Boyraz, Neha Nagpal
Keywords(s): Aging, dyskeratosis congenita, Telomere
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Therapeutics, Technology Classifications > Invention Type > Therapeutics > Small Molecule, Technology Classifications > Invention Type > Therapeutics > RNA (ie. mRNA, siRNA)
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Innovative Therapeutic Approach for Treating Thrombocytopenia Using DNA Damage Repair Inhibitors
Innovative Therapeutic Approach for Treating Thrombocytopenia Using DNA Damage Repair Inhibitors
Background
Thrombocytopenia is a dangerous condition where the number of platelets in the blood is too low (less than 150 billion per liter), increasing the risk of serious bleeding. This condition may arise in certain immune disorders after chemotherapy,...
Published: 3/30/2026
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Updated: 12/5/2025
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Inventor(s): Joseph Italiano, Kellie Machlus, Roelof Bekendam, Andrew Stone
Keywords(s): Thrombocytopenia
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Novel Targets
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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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Targeting ZNF410 for fetal hemoglobin induction in beta-hemoglobinopathies
Targeting ZNF410 for fetal hemoglobin induction in beta-hemoglobinopathies
Background
β-hemoglobinopathies, such as sickle cell disease and β-thalassemias, are genetic disorders characterized by defective hemoglobin production, resulting in anemia and impaired oxygen delivery. These conditions affect millions worldwide, posing significant...
Published: 3/26/2026
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Updated: 11/29/2025
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Inventor(s): Daniel Bauer, Divya Vinjamur
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Novel Targets
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Targeting prosaposin (pSAP) to dendritic cells as novel cancer immunotherapy
Targeting prosaposin (pSAP) to dendritic cells as novel cancer immunotherapy
Background
Antigen-specific T cell responses are central to protection against cancer. Abundance and proper function of immune cells, including lymphocytes and antigen-presenting cells such as dendric cells (DCs), at the tumor site are vital for effective immunity and control...
Published: 3/23/2026
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Updated: 11/29/2025
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Inventor(s): Florian Winau
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Disease Area > Oncology, Technology Classifications > Invention Type > Novel Targets
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(3284, 4360) Niche endothelial cell technology to create red blood stem cells
Engineering Endothelial Niche Cells for Enhanced HSPC Expansion and Transplantation
Background
Haematopoietic stem and progenitor cells (HSPCs) offer a curative potential for various blood and immune diseases through transplantation. Their success, however, is limited by the scarcity of immune-matched donors. Efforts in in vitro expansion of HSPCs...
Published: 3/6/2026
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Updated: 11/4/2025
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Inventor(s):
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Therapeutics > Cell Therapy
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Using defined factors to engineer niche endothelial cells for growing and maintaining blood stem cells
Use of engineered niche endothelial cells to grow and maintain blood stem cells
Background
Hematopoietic stem and progenitor cells (HSPCs) are a rare cell population found in bone marrow that are responsible for producing all blood and immune cells. These cells are especially valuable for their ability to regenerate the entire blood system after transplantation...
Published: 3/25/2026
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Updated: 11/4/2025
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Inventor(s): Leonard Zon, Elliott Hagedorn
Keywords(s):
Category(s): Technology Classifications > Disease Area > Hematology/Oncology, Technology Classifications > Invention Type > Therapeutics > Cell Therapy
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