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BioWorld - Tuesday, December 23, 2025
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Home » Topics » BioWorld Science, Hematologic

BioWorld Science, Hematologic
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Liver
Hematologic

Poseida Therapeutics advances nonviral liver-directed gene therapies

April 18, 2024
Poseida Therapeutics Inc. has announced progress with its fully nonviral liver-directed gene therapies.
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Red blood cells, DNA
Hematologic

NHLBI grant supports Geneventiv’s development of universal gene therapy for hemophilia

April 17, 2024
Geneventiv Therapeutics Inc. has been awarded a Direct to Phase II Small Business Innovation Research (SBIR) grant for approximately $2.5 million from the National Heart Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH) to support development of a universal gene therapy for hemophilia A or B with or without inhibitors.
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Bloodstream with red and white blood cells and platelets
Hematologic

PAR4 antagonist demonstrates robust antithrombotic efficacy in vivo

April 12, 2024
Researchers from Bristol Myers Squibb Co. have published details on the discovery and characterization of novel protease-activated receptor 4 (PAR4) antagonists as potential antiplatelet therapy candidates.
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Blood clot under microscope.
Hematologic

Cereno Scientific submits CTA in Europe for phase I study of new antithrombotic treatment

April 11, 2024
Cereno Scientific AB has submitted a clinical trial application (CTA) to the EMA seeking approval for a first-in-human, phase I study of CS-014, a novel histone deacetylase (HDAC) inhibitor drug candidate under development as an antithrombotic treatment.
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AI-generated image of blood cells in a bone marrow biopsy
Hematologic

Iron rheostat turns on blood stem cells

Feb. 27, 2024
By Mar de Miguel
Iron regulates the metabolism of hematopoietic stem cells (HSCs) and acts as a genetic control of their fate, preserving their identity and regenerative capacity during tissue maintenance and repair. A group of scientists at Albert Einstein College of Medicine has described the key components of a molecular pathway that iron regulates. “What we are proposing here with this mechanism is that iron serves like a switchboard and a sensor,” senior author Britta Will told BioWorld. Will is at the Department of Oncology, the Cell Biology Department, and the Ruth and David Gottesman Institute for Stem Cell Research and Regenerative Medicine at Albert Einstein College of Medicine.
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Illustration of blood vessel that isn't clotting due to hemophilia
Hematologic

siRNA strategy may help in long-term control of hemophilia

Feb. 26, 2024
By Coia Dulsat
In a paper published on Feb. 21, 2024, in Science Translational Medicine, researchers from the Versiti Blood Research Institute described how they successfully controlled bleeding for months in hemophilia A models using a siRNA therapy that targeted plasminogen.
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Hematologic

Ildong Pharmaceutical patents ANO6 blockers for treatment of thrombosis

Feb. 19, 2024
Ildong Pharmaceutical Co. Ltd. have synthesized heteroaryl derivative compounds acting as anoctamin-6 (ANO6; TMEM16F) blockers.
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Hematologic

CBP/p300 HAT domain inhibitor rapidly expands blood leukocyte compartment in vivo

Feb. 19, 2024
In a recent study, researchers from Technische Universität Dresden and affiliated organizations evaluated the novel small-molecule inhibitor of the CBP/p300 HAT domain, A-485, as a potential therapeutic candidate for the treatment of hematological cancers and other pathologies related to blood leukocyte compartment.
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Biomarkers

New COL3A1 mutation found in case of bleeding of unknown cause

Feb. 12, 2024
Bleeding of unknown cause is a group of rare disorders that are still difficult to accurately diagnose. A case report on a patient with hematoma in the perineal region after her first delivery was presented.
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Biomarkers

Novel heterozygous missense mutation in the FGG gene linked to hypodysfibrinogenemia

Feb. 12, 2024
The rare hereditary fibrinogen disorder hypodysfibrinogenemia is characterized by fibrinogen defects, which can cause thrombotic and hemorrhagic phenotypes that are not always predicted by routine coagulation tests. Researchers from Academic Hospital Maastricht aimed to characterize the genetic profile of a family with hypodysfibrinogenemia and predict bleeding and/or thrombotic phenotypes in asymptomatic family members using innovative testing.
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