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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Handshake with DNA, molecules

Orum scores potential $945M deal with Vertex for novel DACs

July 16, 2024
By Marian (YoonJee) Chu
Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel).
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Microbiome illustration
Hematologic

Phage-derived enzyme can help fight acute graft-vs.-host disease

July 16, 2024
By Tamra Sami
Researchers in Japan have discovered that a phage-derived enzyme called endolysin, which targets highly resistant biofilm-forming bacteria, could help restore the gut microbiota to mitigate acute graft-vs.-host disease. Acute graft-vs.-host disease (aGVHD) is a common complication for patients who undergo allogeneic hematopoietic cell transplantation (allo-HCT). Recent studies have highlighted the significant role of the microbiome in aGVHD, with dysbiosis contributing to its pathogenesis.
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Illustration of clot forming in blood vessel
Cardiovascular

Antibody shows potential in thrombotic thrombocytopenic purpura

July 8, 2024
Von Willebrand factor (vWF) plays a crucial role in hemostasis, and elevated levels are associated with risk of thrombosis. Gain-of-function mutations in the C-domain of vWF are linked to an increased risk of thrombosis. Previous work has shown that disrupting cysteine-rich C-domain in vWF led to reduced platelet recruitment in a shear-dependent fashion, making it a promising target for pharmacology. Apollo Therapeutics Ltd. has presented data on antibodies targeting the C-domain of vWF, including AP-21 as antithrombotics.
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Illustration of blood vessel that isn't clotting due to hemophilia
Hematologic

SS-315 shows hemostatic effects in mice with hemophilia A

July 4, 2024
Beijing Gensciences Inc. has developed a FVIIIa-mimetic bispecific antibody named SS-315 for the treatment of hemophilia A. SS-315 was developed by targeting FX with its upper Fab arms and FIXa with its down-side scFv arms, respectively; the hemostatic potential of SS-315 was investigated in vitro and in vivo.
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Stroke illustration: brain, artery, neurons
Neurology/psychiatric

GPVI inhibitor EMA-601 has unprecedented potency in preclinical ischemic stroke

July 3, 2024
Glycoprotein VI (GPVI) is a platelet collagen receptor involved in platelet activation and an emerging target for treating thrombotic disorders such as ischemic stroke. Data have been presented by University of Würzburg scientists regarding a humanized anti-GPVI Fab antibody, EMA-601, with unprecedented potency in vitro and in vivo.
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Syringe, vial with pound currency symbol

UK agrees to payment scheme for costly gene therapy Hemgenix

June 28, 2024
By Nuala Moran
CSL Behring’s expensive hemophilia B gene therapy is to be reimbursed by the U.K. National Health Service, after the company agreed to an outcomes-based payment scheme. The therapy, Hemgenix (etranacogene dezaparvovec), which has a U.K. list price of £2.6 million (US$3.3 million), was approved under a managed access scheme, in which data will be collected over five years to enable both the long-term effectiveness, and any adverse liver toxicity caused by the transgene, to be monitored.
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Pink and orange test tubes
Hematologic

Targed Biopharmaceuticals’ TGD-001 attenuates thrombocytopenia and tissue damage in TTP

June 27, 2024
The heterogenous composition of thrombi limits the efficacy of existing therapies.
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Biomarkers

Study unveils rare variant in ENG gene tied to venous thrombosis

June 27, 2024
There is growing evidence of the role of soluble endoglin in the biology of platelets, including thrombosis. French researchers have investigated the role of genetic variants in the gene encoding endoglin, ENG, and the risk of venous thrombosis development.
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Hematologic

REGN-7999 reverses liver iron overload, improves bone health in mouse model of beta-thalassemia

June 26, 2024
Transmembrane serine protease 6 (TMPRSS6) is a negative regulator of hepcidin, which is the main iron homeostasis-regulating hormone. Regeneron Pharmaceuticals Inc. has recently presented preclinical data for the monoclonal antibody targeting TMPRSS6, REGN-7999, which is being developed for the treatment of iron overload.
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Takeda moves mezagitamab to phase III in thrombocytopenia

June 25, 2024
By Tamra Sami
Takeda Pharmaceutical Co. Ltd. is progressing mezagitamab to phase III trials after the CD38 monoclonal antibody showed rapid and sustained increases in platelet counts in patients with persistent or chronic primary immune thrombocytopenia (ITP) in a phase IIb trial.
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