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BioWorld - Saturday, May 9, 2026
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Acute myeloid leukemia illustration
Cancer

Bristol Myers Squibb presents a molecular glue degrader for leukemia treatment

Sep. 3, 2024
Genetic or pharmacologic inhibition of casein kinase 1α (CK1α) has proven effective in suppressing the proliferation of acute myeloid leukemia (AML) cell lines with wild-type TP53. Researchers from Bristol Myers Squibb Co. recently presented the discovery and preclinical characterization of BMS-986397, a cereblon E3 ligase modulatory drug (CELMoD) designed as a molecular glue degrader of CK1α.
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Cardiovascular

KUS-121 shows therapeutic potential in models of atherosclerotic disease

Sep. 3, 2024
The valosin-containing protein (VCP) modulator KUS-121 from Kyoto University is in phase II development as an intravitreal treatment for nonarteritic central retinal artery occlusion, but its efficacy in atherosclerosis is unknown.
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Lung cancer illustration
Cancer

Novel EGFR inhibitor overcomes common resistances to NSCLC treatments

Sep. 3, 2024
Researchers from Nankai University reported on [I], a novel EGFR L858R/T790M/C797S inhibitor designed to overcome the most frequent resistances to non-small-cell lung cancer (NSCLC) treatment.
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Cardiovascular

hERG channel agonists show promise for treating long QT syndrome type 8

Sep. 3, 2024
Repolarization abbreviation with the antiarrhythmic drug mexiletine has been found to normalize the QTc interval and reduce the rate of events in long QT syndrome type 3 (LQT3), but it is only partially efficacious in other malignant forms, including LQT2 and LQT8. There are compounds that have not been tested, such as hERG channel agonists, which are capable of shortening repolarization. The potent hERG agonist ICA-105574 was thus tested in a clinically relevant porcine model of LQT8.
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3D illustration of T cells fighting cancer
Immuno-oncology

Highfield Biopharmaceuticals files IND in China for immunoliposome HF-50 for solid tumors

Sep. 3, 2024
Highfield Biopharmaceuticals Inc. has filed an IND application with China’s National Medical Products Administration (NMPA) to conduct a clinical trial of HF-50, an immunoliposome that directs T cells to attack solid tumors and enhances anticancer activity with an immune modulator.
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Alpha-galactosidase enzyme
Endocrine/metabolic

GC Biopharma and Hanmi’s once-monthly subcutaneous treatment for Fabry disease gains US IND clearance

Sep. 3, 2024
The FDA has cleared an IND for a phase I/II trial of LA-GLA (GC-1134A, HM-15421), an innovative enzyme replacement therapy for Fabry disease being co-developed by GC Biopharma Corp. and Hanmi Pharmaceutical Co. Ltd.
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3D illustration of acute lymphoblastic leukemia in blood.
Immuno-oncology

Hemogenyx develops improved version of CDX for leukemia

Sep. 3, 2024
Hemogenyx Pharmaceuticals plc has developed a new and improved version of its bispecific antibody CDX for the treatment of relapsed/refractory acute myeloid leukemia, a subset of acute lymphoblastic leukemia, and potentially for conditioning in bone marrow transplantations.
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Heart illustration
Cardiovascular

Ghrelin receptor agonist improves cardiac function in preclinical heart failure

Sep. 3, 2024
At the recent European Society of Cardiology meeting, Anacardio AB and Helsinn Healthcare SA presented data on their oral small-molecule ghrelin receptor agonist and first-in-class calcium sensitizing inotrope, AC-01, in preclinical models of heart failure.
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Cancer

UBR1 as therapeutic target for anaplastic thyroid carcinoma

Sep. 3, 2024
Scientists from Wuhan University and affiliated organizations have published findings from their investigation into the role of Yes-associated protein (YAP), which is a critical effector of the Hippo pathway, in anaplastic thyroid carcinoma.
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Illustration of tau proteins in Alzheimer's disease
Neurology/psychiatric

RING-nanobody degraders clear and prevent aggregation in tauopathy models

Sep. 3, 2024
By Coia Dulsat
Researchers from the UK Dementia Research Institute at the University of Cambridge have found how to prevent and reverse tau aggregation using target-specific nanobodies. The team holds great expertise in the role of TRIM21 in the tau environment since William McEwan, senior author of the study, first discovered TRIM21 and, a bit later, defined its contribution to tau immunotherapy efficacy.
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