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BioWorld - Tuesday, December 23, 2025
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AI-generated image of blood cells in a bone marrow biopsy
Cancer

Eilean’s ZE74-0282 shows promise in JAK2 V617 mutant diseases

Dec. 23, 2025
No Comments
Eilean Therapeutics LLC has presented data for ZE74-0282, a novel small molecule that targets the JAK2 JH2 domain harboring the V617F mutation with no impact on wild-type JAK2.
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Multiple myeloma illustration
Immuno-oncology

LBL-076: a first-in-class trispecific TCE for refractory MM

Dec. 23, 2025
No Comments
Despite therapeutic advances, multiple myeloma (MM) remains incurable, with most patients relapsing and developing resistance, especially those refractory to proteasome inhibitors, immunomodulatory drugs and anti-CD38 antibodies. Limited options and poor prognosis highlight the need for new agents with distinct mechanisms and better safety.
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Immuno-oncology

Crossbow Therapeutics highlights activity of TERT-targeting CBX-663

Dec. 23, 2025
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Crossbow Therapeutics Inc. has provided details on the preclinical characterization for the telomerase reverse transcriptase (TERT)-targeting T-cell engager CBX-663.
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Illustration of red blood cells traveling in the arteries
Hematologic

DXO-1801 shows promise for inflammation-driven anemia

Dec. 22, 2025
No Comments
Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.
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Blood samples in lab
Cancer

AJ1-11095 outperforms ruxolitinib in myeloproliferative neoplasm models

Dec. 22, 2025
No Comments
JAK2 inhibitors (JAK2i) are the standard treatment for myelofibrosis (MF), offering symptom relief and reducing spleen size. However, all FDA-approved JAK2i are type I inhibitors, which fail to eliminate the mutant MPN clone, leading many patients to treatment discontinuation.
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Sickle cell disease 3D illustration
Hematologic

EMU-116 performs better than plerixafor in sickle cell disease

Dec. 19, 2025
No Comments
Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.
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Cancer

ONC-206 shows activity in TNBC models

Dec. 19, 2025
No Comments
Researchers from Chimerix, now part of Jazz Pharmaceuticals, presented preclinical data on ONC-206, a compound that functions as both an agonist of the mitochondrial protease Caseinolytic peptidase P (CLPP) and an antagonist of the G protein-coupled receptor DRD2, in models of triple-negative breast cancer (TNBC).
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Cancer and blood cells
Hematologic

AND-017 ameliorates anemia in MDS mice

Dec. 18, 2025
No Comments
Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.
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Cancer

ATNM-400 radioconjugate shows efficacy in resistant breast cancer

Dec. 18, 2025
No Comments
Although hormone receptor-positive (HR+) breast cancer accounts for over 70% of cases, 20%-30% of patients still experience relapse despite endocrine therapies such as tamoxifen. Recurrence is also observed in HER2+ breast cancer, even with HER2-targeted antibodies and antibody-drug conjugates (ADCs), the use of which can be limited by adverse effects such as interstitial lung disease.
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Test tubes, dropper
Immuno-oncology

MDX-2003 exerts potent activity in preclinical B-cell malignancies

Dec. 17, 2025
No Comments
Modex Therapeutics Inc. has developed MDX-2003, a first-in-class tetraspecific T-cell engager targeting CD19 and CD20 on B cells, while co-engaging CD3 and CD28 on T cells.
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