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BioWorld - Tuesday, July 28, 2026
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Home » Topics » BioWorld Science, Hematologic

BioWorld Science, Hematologic
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DNA double helix under a magnifying glass
Hematologic

Financing at Immitra Bio to advance in vivo gene editing therapies

July 22, 2026
No Comments
Immitra Bio GmbH has completed a CHF2.4 million (US$ 3 million) pre-seed financing round as it advances development of a platform for scalable, one-time curative in vivo gene editing therapies based on its mutation-agnostic gene editing technology and digital target identification platform.
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Red CAR T cell on blue blackground
Drug design, drug delivery & technologies

To viral or not to viral: In vivo CAR T debates delivery tech

July 21, 2026
By Marian (YoonJee) Chu
No Comments
The field of cell and gene therapy is moving toward a new phase of scalability and clinical durability, with in vivo chimeric antigen receptor (CAR) T-cell therapeutics emerging as the dark horse in what speakers at BIO Asia-Taiwan 2026 described as CAR T’s second revolution.
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Illustration of tumor
Immuno-oncology

Vascular endothelial cells can present tumor antigens

July 21, 2026
By Anette Breindl
No Comments
Investigators at the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) have demonstrated that in the liver, some endothelial cells (ECs) acted as antigen-presenting cells of sorts. These lipoprotein lipase (LPL)-expressing cells cross-presented tumor antigens on their surface, alerting T cells to the presence of metastases.
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Hematologic

HMB-003 outperforms tranexamic acid in fibrinolysis models

July 16, 2026
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Excessive fibrinolysis is a major driver of bleeding across multiple clinical settings, including heavy menstrual bleeding and other hemorrhagic disorders. Researchers from Hemab ApS presented preclinical data on HMB-003, a long-acting plasmin inhibitor that directly targets the enzyme’s active site to achieve sustained antifibrinolytic activity.
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Hematologic

Preclinical data support continued development of TGM-148 for bleeding disorders

July 15, 2026
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The prophylaxis of bleeding disorders usually requires frequent intravenous infusions that result in excessive treatment burden and residual bleeding risk. TGM-148 is a siRNA developed by Tangram Therapeutics plc designed to repress the hepatocyte expression of a gene target to rebalance hemostasis, and has shown efficacy in murine models of hemophilia and von Willebrand disease.
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Hematologic

JAK2 inhibitors reported in Prelude Therapeutics patent

July 13, 2026
Prelude Therapeutics Inc. has identified new tyrosine-protein kinase JAK2 inhibitors potentially useful for the treatment of leukemia, polycythemia vera, myelofibrosis, essential thrombocythemia and graft-vs.-host disease.
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Drug design, drug delivery & technologies

ARPA-H program supports advances for rare genetic diseases

July 10, 2026
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The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services, has announced the teams for the THRIVE (Treating Hereditary Rare diseases with In Vivo prEcision genetic medicines) program. With a commitment of up to $160 million over 5 years, THRIVE aims to accelerate solutions for rare genetic pediatric diseases across multiple technological approaches, clinical trial designs and deployment models.
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Illustration of red and white blood cells in an artery
Biomarkers

Case report links APOLD1 variant to vascular-type bleeding disorder

June 23, 2026
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Vascular-type bleeding disorder (BDVAS) is a rare, autosomal dominant disorder mainly caused by impaired vascular integrity.
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Photo of pipette with test tube
Hematologic

Preclinical data on ALN-CFB for paroxysmal nocturnal hemoglobinuria presented

June 22, 2026
No Comments
The alternative pathway (AP) of the complement system is crucial for innate immunity and its dysregulation may lead to several diseases, including paroxysmal nocturnal hemoglobinuria (PNH). Alnylam Pharmaceuticals Inc. and Regeneron Pharmaceuticals Inc. recently presented data on ALN-CFB, a siRNA targeting hepatic complement factor B (CFB) that inhibits the production of CFB in the liver.
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Red blood cells, DNA
Hematologic

For clonal hematopoiesis, epigenetics can be in driver’s seat

June 15, 2026
By Anette Breindl
No Comments
Clonal hematopoiesis (CH), where few blood stem cells produce a significant fraction of mature blood cells that are genetically identical, is partly an inevitable feature of aging. Certainly, it is near universal in those older than 60. CH is not itself a disease, but 1%-2% of CH cases progress to acute myeloid leukemia, and it raises the risk of some other types of cancer as well. A total of eight genes are responsible for 95% of CH cases, George Vassiliou told the audience in Saturday’s plenary session at the 2026 Annual Congress of the European Hematology Association (EHA 2026).
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