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BioWorld - Saturday, April 11, 2026
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Mouse genome/DNA sequencing concept art.
Genetic/congenital

Researchers develop novel Kabuki syndrome murine model

March 7, 2025
Lysine demethylase 6A (KDM6A) is a demethylase that plays a key role at regulating developmental gene expression signatures in several tissues, including neuronal cells. The KDM6A gene is located in chromosome X and pathogenic variants in this gene are tied to Kabuki syndrome type 2. Even though progress in understanding the functions of KDM6A has been made, its role in cochlear development and auditory function remains poorly understood.
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Antibodies attacking cancer cell
Immuno-oncology

VCR-036, a pentavalent bispecific PD-1/CTLA4 Vincobody with promising preclinical safety and efficacy

March 7, 2025
Researchers from Vicero Inc. have developed a Vincobody platform, which allows for design of novel proprietary VHH antibody fragments that possess the efficacy of dual checkpoint blockade while mitigating the toxicity limitations of current monoclonal antibody therapies.
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Cancer

Novel HDAC3 inhibitor with high oral availability divulged

March 7, 2025
Histone deacetylase 3 (HDAC3) is involved in transcriptional regulation, phosphorylation and the inhibition of tumor suppressor genes. Its upregulation in several types of tumors, such as colorectal, prostate, breast and ovarian cancers, among others, makes it a potential therapeutic target in cancer.
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Light micrograph and 3D illustration of adipose tissue.
Endocrine/metabolic

ARO-ALK7 demonstrates safety and efficacy in obesity mouse model

March 7, 2025
Previous research has demonstrated that activin receptor-like kinase 7 (ALK7) signaling suppresses lipolysis, resulting in increased adipocyte size and lipid content. Additionally, predicted loss of function variants in the ALK7 gene (ACVR1C) have been associated with reduced waist-to-hip ratio (WHR) adjusted for body mass index, protection from type 2 diabetes, as well as reduced risk of cardiovascular disease.
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Dendritic cells
Neurology/psychiatric

GluN2B signaling regulates dendritic spine plasticity and reverses FXS phenotypes

March 7, 2025
Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and a leading monogenic cause of autism, yet effective treatments remain elusive. Previous work showed that N-methyl-D-aspartate receptors (NMDARs) play a prominent pathophysiological role in FXS and other neurodevelopmental disorders.
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Neurology/psychiatric

NEK2 inhibition improves neurological function in model of multiple sclerosis

March 7, 2025
Researchers from General Hospital of Tianjin Medical University presented data from a study that aimed to explore the role of NIMA-related kinase 2 (NEK2) in regulating B-cell immunity in autoimmune diseases. It was demonstrated that NEK2 is highly expressed in patients with multiple sclerosis.
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Cancer

Accent Therapeutics discovers new DHX9 inhibitors

March 6, 2025
Accent Therapeutics Inc. has described ATP-dependent RNA helicase A (DHX9) inhibitors reported to be useful for the treatment of cancer.
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Inflammatory

NLRP3 inflammasome inhibitors disclosed in Transthera patent

March 6, 2025
Transthera Biosciences Co. Ltd. has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of autoimmune diseases and inflammatory disorders.
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Cancer

Abbisko Therapeutics describes new EGFR mutant inhibitors

March 6, 2025
Abbisko Therapeutics Co. Ltd. has identified EGFR mutant inhibitors reported to be useful for the treatment of cancer.
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Infection

Merck Sharp & Dohme divulges new viral replication inhibitors

March 6, 2025
Merck Sharp & Dohme LLC has synthesized viral replication inhibitors reported to be useful for the treatment of respiratory syncytial virus and metapneumovirus infections.
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