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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
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Red blood cells illustration.
Hematologic

RNA Therapeutics obtains pre-IND guidance from FDA on RNAT-89

Aug. 20, 2024
RNA Therapeutics Inc. has received a written response from the FDA to its pre-IND application questions regarding the development of its lead asset, RNAT-89 (BLA-761423), an mRNA LNP formulation to express darbepoetin.
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Illustration of sickle-cell and normal red blood cells
Hematologic

Long-term safety study for sickle cell anemia gene therapy

Aug. 8, 2024
A recent study in PLoS One by researchers from Cincinnati Children’s Hospital Medical Center has evaluated the preclinical long-term safety of human A gamma-globin gene-carrying GbGM LV in wild-type mice.
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Hematologic

WIZ glue degrader restores fetal hemoglobin

Aug. 5, 2024
Scientists at the Novartis Institutes for Biomedical Research (NIBR) in Cambridge have discovered a small molecule that could be used as a therapy for sickle cell disease (SCD). The molecular glue oral degraders of the WIZ transcription factor called dWIZ-1 and dWIZ-2, bind to cereblon (CRBN) and WIZ, marking it for degradation and inducing the expression of fetal hemoglobin (HbF).
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CAR T cell attacking cancer cells
Cancer

Removing CD5 'brake' on CAR T cells increases antitumor efficacy

Aug. 2, 2024
By Mar de Miguel
To be successful, CAR T-cells need a balance between being effective and overkill. Researchers from the University of Pennsylvania and Vittoria Biotherapeutics Inc. have eliminated the CD5 signaling pathway of their CAR Ts to prevent the immunosuppressive brake effect. In return, this improved their proliferation and antitumor activity in T cell lymphomas.
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Sickle cell illustration
Hematologic

Actinium Pharmaceuticals obtains FDA clearance for trial of Iomab-ACT in sickle cell disease

July 26, 2024
Actinium Pharmaceuticals Inc. has received FDA clearance of an IND application to study Iomab-ACT for targeted conditioning prior to a bone marrow transplant (BMT) in patients with sickle cell disease. 
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FDA website and logo

FDA approves second Soliris biosimilar

July 23, 2024
By Marian (YoonJee) Chu
Samsung Bioepis Co. Ltd., of Incheon, South Korea, gained U.S. FDA approval of Epysqli (eculizumab-aagh) as the second biosimilar product to Alexion Pharmaceuticals Inc.’s Soliris (eculizumab) to treat two rare diseases. The regulatory clearance July 22 grants use of Epysqli to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome – two rare hematologic- and kidney-related disorders known to affect about 50,000 and 5,000 patients in the U.S., respectively.
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HHS posts negative opinion on Bluebird’s fertility support program

July 23, 2024
The U.S. Department of Health and Human Services’ Office of the Inspector General disclosed an advisory opinion finding Bluebird Bio Inc.’s fertility support program for a gene therapy treatment could run afoul of federal anti-kickback statutes. That follows a similar opinion against Vertex Pharmaceuticals Inc., and its fertility program associated with gene-editing therapy Casgevy (exagamglogene autotemcel). Vertex subsequently filed a lawsuit.
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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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