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Home » Topics » Disease categories and therapies » Hematologic

Hematologic
Hematologic RSS Feed RSS

Pfizer pulls sickle cell drug after deaths and health complications

Sep. 26, 2024
By Lee Landenberger
The risk and benefit of Pfizer Inc.’s oral sickle cell disease drug Oxbryta (voxelotor) has flipped, prompted by what the company called new clinical data indicating “an imbalance in vaso-occlusive crises and fatal events” that need more study. Based on an EMA recommendation, Pfizer said it is voluntarily recalling all lots of Oxbryta from wherever it’s approved worldwide. Pfizer also is shuttering its Oxbryta clinical studies and expanded access programs.
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EMA icons

EMA’s CHMP recommends Hympavzi for hemophilia, Elahere for cancer

Sep. 23, 2024
By Nuala Moran
For once, the EMA appears to have pipped the U.S. FDA to the post, with Pfizer Inc.’s hemophilia A and B therapy Hympavzi (marstacimab) recommended for approval in Europe on Sept. 20, while the U.S. PDUFA date is set for the fourth quarter of the year.
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Blood sample, DNA
Hematologic

Metagenomi reports preclinical data supporting development of MGX-001 for hemophilia A

Sep. 4, 2024
Metagenomi Inc. has reported data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001.
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Illustration of zebrafish, gene editing.
Hematologic

Zebrafish lacking Dnajc21 show cytopenia and impaired nucleotide metabolism

Aug. 30, 2024
Shwachman-Diamond syndrome is an inherited bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, cytopenia, growth restriction and skeletal abnormalities, and for which primary treatment is allogeneic hematopoietic stem cell transplantation, which is associated with significant toxicity.
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Sickle cells
Hematologic

Targeting PGC-1α as therapeutic strategy against sickle cell disease

Aug. 26, 2024
Recent studies have shown that up-regulation of peroxisome proliferator-activated receptor γ coactivator-1α (PGC-1α) was able to induce fetal hemoglobin synthesis in human primary erythroblasts.
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Hematologic

New PHD2 inhibitors disclosed in Oxford University Innovations patent

Aug. 22, 2024
Oxford University Innovations Ltd. has divulged Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of anemia, ischemia, cardiovascular disorders, inflammatory disorders, irritable bowel syndrome, chronic kidney disease, Parkinson’s disease and Alzheimer’s disease, among others.
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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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