The FDA has cleared Prime Medicine Inc.’s IND application for PM-359 for the treatment of chronic granulomatous disease (CGD), enabling initiation of a phase I/II trial in the U.S.
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.
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.
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.
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.
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.
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.
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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