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BioWorld - Monday, December 15, 2025
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Red blood cells on blue background

ASH 2024: Agios’ mitapivat checks off efficacy; liver toxicity a concern

Dec. 9, 2024
By Brian Orelli
Agios Pharmaceuticals Inc. is learning the hard way the downside of having a pipeline in a product. When a side effect crops up, investors are likely to worry that it may affect the potential of the drug in the numerous diseases the drug could potentially treat.
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Cancer

Researchers develop humanized xenograft glioblastoma model in mice

Dec. 9, 2024
Researchers have described the development of a novel murine model of glioblastoma established with a patient-derived xenograft (PDX) in humanized mice that harbored an almost complete human immune microenvironment.
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Drug R&D concept image.
Immuno-oncology

ALT-100 shows antitumoral glioma activity by NAMPT neutralization

Dec. 9, 2024
Aqualung Therapeutics Corp. has tested ALT-100.
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Non-Hodgkin lymphoma cells in the blood flow
Immuno-oncology

JNJ-87801493 enhances T-cell engager efficacy in B-NHL

Dec. 9, 2024
Researchers from Janssen Research & Development LLC presented preclinical data for JNJ-87801493, a first-in-class CD20 targeted CD28 costimulatory bispecific antibody (Ab), currently in early clinical development for the treatment of B-cell malignancies.
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Jun R. Huh at Healthcare Innovation Forum 2024
HIF 2024

Immune system could be key to neurodegenerative disease

Dec. 5, 2024
By Marian (YoonJee) Chu
Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis.
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Liver and DNA
Endocrine/metabolic

New siRNA therapy candidate, ETX-312, shows efficacy in MASH mouse model

Dec. 5, 2024
Researchers from E-Therapeutics plc presented preclinical data for ETX-312, a GalNAc-conjugated short interfering RNA (siRNA), produced using the Galomic platform and being developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
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Cancer

TAT-Cx43(266-283) shows potential for treating glioblastoma multiforme

Dec. 5, 2024
Glioblastomas (GBMs) are the most malignant brain tumors and remain uncurable. Even after therapy, some glioblastoma stem cells (GSCs) survive in the brain parenchyma and cause tumor recurrence.
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Cancer

New murine glioblastoma model developed by University of Toronto

Dec. 5, 2024
A team of investigators at the University of Toronto have developed a new murine model.
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Cancer

DNA-damaging small molecule shows efficacy against MMR-deficient recurrent gliomas

Dec. 5, 2024
Researchers from Northwestern University and Yale University have presented new preclinical data for KL-50, a small molecule under investigation for the treatment of glioblastoma. KL-50 was designed to fluoro-ethylate DNA bases, resulting in DNA inter-strand cross-links and subsequent DNA damage that triggers DNA mismatch repair (MMR)-independent apoptosis.
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Hepatitis B virus
Infection

SA-1211, a dual-target siRNA with promising antiviral activity in AAV-HBV mice

Dec. 4, 2024
Researchers from Suzhou Siran Biotech Co. Ltd. presented the discovery and preclinical characterization of SA-1211, an N-acetylgalactosamine (GalNAc)-conjugated siRNA dimer targeting both hepatitis B virus (HBV) and PD-L1 gene expression, being developed as a potential new therapeutic candidate for the treatment of chronic hepatitis B (CHB).
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