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BioWorld - Thursday, December 25, 2025
Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Hematologic

TFPI potentially useful as target for treatment of undiagnosed bleeding disorders, researchers unveil

Feb. 14, 2023
Undiagnosed bleeding disorders put people at risk due to bleeding without an optimal treatment strategy. Spanish researchers from the Hospital Universitario La Paz and Universidad Autónoma de Madrid have focused on the targeting of tissue factor pathway inhibitor (TFPI) as a potential approach for this medical need by using TFPI blocker antibodies.
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Yen-Yuan currency symbol

Micot targets anticoagulant breakthrough with $15M round

Feb. 13, 2023
By Doris Yu
New drug technology may soon deliver a breakthrough to eliminate internal bleeding caused by drug overuse. Shaanxi Micot Technology Co. Ltd.’s MT-1011 is a synthetic water-soluble small-molecule anticoagulant antagonist. After a single intravenous dose, it can bind directly to other anticoagulants to stop anticoagulant effects and restore the clotting function. Data from clinical trials support its mechanism.
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Biomarkers

NBEAL2 gene mutation tied to gray platelet syndrome with immune deficiency

Feb. 13, 2023
Gray platelet syndrome is an autosomal recessive platelet disorder characterized by macrothrombocytopenia and deficiency or decreased levels of alpha granules that confer a grayish appearance to the platelets. The genetic cause is located at chromosome locus 3p21, affecting the NBEAL2 gene.
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Patient in hospital bed

Vivacelle completes its phase IIa in raising blood pressure

Feb. 10, 2023
By Lee Landenberger
Privately held Vivacelle Bio Inc. has completed its phase IIa study of VBI-S in treating septic shock patients with severe hypovolemia, a drastic drop in blood pressure that can lead to organ failure and death. In the study, VBI-S, an intravenously injected fluid composed of phospholipid nanoparticles, increased blood pressure and improved organ function.
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Illustration of blood vessel that isn't clotting due to hemophilia
Biomarkers

Osteoprotegerin as a biomarker of hemophilia

Feb. 10, 2023

Previous research has suggested that factor VIII (FVIII) can regulate the osteoprotegerin (OPG)/RANKL system, which appears to play a role in hemophilic arthropathy. Investigators have now aimed to measure the OPG levels in patients with hemophilia A/B and assess their correlation with the levels of FVIII/FIX.


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Red blood cells illustration.
Biomarkers

New ITGA2B gene compound heterozygous mutation found in Glanzmann thrombasthenia

Feb. 10, 2023
Glanzmann thrombasthenia (GT) is a rare bleeding disorder caused by defects in the expression of platelet surface integrins, such as integrin alpha-IIb (GPIIb, encoded by ITGA2B).
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Red blood cells

Roche looks to regulators on positive phase III results with rare disease drug

Feb. 7, 2023
By Caroline Richards
Roche Holding AG’s investigational monoclonal antibody, crovalimab, which inhibits part of the innate immune system in patients with the rare blood condition paroxysmal nocturnal haemoglobinuria, met its co-primary efficacy endpoints in a phase III trial that will form the basis of its approval submissions worldwide.
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Cholesterol plaque in artery
Cardiovascular

T-cell study reveals autoimmune component of atherosclerosis

Feb. 2, 2023
By Mar de Miguel
The combination of two sequencing techniques has unveiled features of a subpopulation of cells that could be producing plaques in atherosclerosis. This process is associated with an autoimmune component driven by CD4+ T cells, according to a study from researchers at Leiden University.
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Antibodies and red blood cells

Mabwell out-licenses preclinical iron metabolism MAb to Disc Medicine in $412M deal

Jan. 31, 2023
By Tamra Sami
Just in time for the Chinese New Year, Mabwell Bioscience Co. Ltd. announced that its U.S. subsidiary, Mabwell Therapeutics Ltd., closed a licensing deal with Disc Medicine Inc. worth up to $412 million.
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Cholesterol plaque in artery
Cardiovascular

T-cell study reveals autoimmune component of atherosclerosis

Jan. 31, 2023
By Mar de Miguel
The combination of two sequencing techniques has unveiled features of a subpopulation of cells that could be producing plaques in atherosclerosis. This process is associated with an autoimmune component driven by CD4+ T cells, according to a study from researchers at Leiden University.

“I think that we can clearly say now that atherosclerosis is a very clear autoimmune component. It is a multifactorial disease, a combination of genes, and lifestyle, but also inflammation and the immune system,” Ilze Bot and Bram Slütter, associate professors at the Division of Biotherapeutics of Leiden University, told BioWorld.
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