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BioWorld - Monday, July 13, 2026
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Cancer

St. Jude Children’s Research Hospital presents new modulators of GSPT1 and/or IKZF1 activity

June 22, 2023
St. Jude Children’s Research Hospital has divulged molecular glue degraders comprising cereblon (CRBN) binding agents covalently bound to a eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1) and/or DNA-binding protein ikaros (IKZF1) targeting moiety acting as GSPT1 and/or IKZF1 degradation and GSPT1 and/or IKZF1/CRBN interaction inducers reported to be useful for the treatment of cancer.
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Infection

GSK describes new antimalarial compounds

June 22, 2023
GSK plc has identified pyrazine compounds reported to be useful for the treatment of malaria infection by Plasmodium falciparum.
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Cardiovascular

Novartis divulges new GIRK1/4 IKACh channel blockers

June 22, 2023
Novartis AG has synthesized naphthyridinone derivatives acting as K(ir) 3.1/3.4 (GIRK1/4; KCNJ3/5) IKACh channel blockers reported to be useful for the treatment of bradyrhythmic arrhythmia, bradycardia, atrial fibrillation, heart block, hypotension, primary aldosteronism, sick sinus syndrome and vasovagal syncope, among others.
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Neurology/Psychiatric

Remix Therapeutics patents new RNA splicing modulators

June 22, 2023
Remix Therapeutics has disclosed compounds acting as RNA splicing modulators reported to be useful for the treatment of cancer and Huntington’s disease.
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Antibiotic resistant bacteria inside a biofilm
Infection

CTC-177 well-tolerated with evidence of prophylaxis against multidrug-resistant gram-negative bacteria

June 22, 2023
Having demonstrated in previous work that drug-Fc conjugates (DFCs) are a promising treatment alternative for multidrug-resistant (MDR) gram-negative bacteria, researchers from the Center for Discovery and Innovation and Cidara Therapeutics Inc. presented results from the identification of CTC-177, a novel DFC, as a potential immunoprophylactic agent against MDR gram-negative bacterial infections.
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Pediatric brain illustration
Biomarkers

INDYGON syndrome, a novel spliceosomopathy caused by biallelic variants in SART3

June 22, 2023
Researchers from Murdoch Children’s Research Institute presented data from a study that linked recessive variants in the SART3 gene with a novel neurodevelopmental syndrome.
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Antibodies attacking virus cell
Infection

Researchers engineer a broadly neutralizing antibody pan-hantavirus therapeutic candidate

June 22, 2023
A cluster of deaths in 1993 in the southwestern U.S. that were characterized by an acute respiratory failure and shock were determined to be due to hantaviruses, which are RNA zoonotic viruses typically transmitted by a rodent vector. Given a fatality rate ranging from 15% to 40% and capacity to potentially spread through human-to-human contact, it is possible that the public health risks posed by these agents have been underestimated.
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Illustration of intestines with inflammation
Gastrointestinal

Mozart Therapeutics’ bispecific CD8 Treg modulator does not increase unwanted immune cell activation, pro-inflammatory cytokines

June 22, 2023
Mozart Therapeutics Inc. has reported preclinical pharmacologic and tolerability data for MTX-101, a bispecific CD8 regulatory T-cell (Treg) modulator targeting inhibitory KIR2DL(1/2/3) and CD8 expressed on CD8 Tregs. The autoimmune checkpoint inhibitor aims to restore CD8 Treg function, acting early in the autoimmune disease process to halt downstream inflammation and prevent further complication. The initial therapeutic focus for MTX-101 is gastrointestinal autoimmune disorders.
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Liver disease
Gastrointestinal

HPG-7233 demonstrates promising PK and efficacy in preclinical model of NASH

June 22, 2023
At the ongoing EASL meeting, researchers from Hepagene Therapeutics Inc. presented preclinical data for the novel thyroid hormone receptor β (THR-β) agonist HPG-7233, being developed for the treatment of nonalcoholic steatohepatitis (NASH).
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Y chromosomes and DNA
Cancer

Studies give new insights into role of Y chromosome and its denizens in cancer progression

June 22, 2023
By Anette Breindl
Back-to-back papers in the June 22, 2023, issue of Nature have identified separate molecular mechanisms underlying sex-specific cancer outcomes. Researchers from The University of Texas MD Anderson Cancer Center showed that increased expression of the epigenetic enzyme KDM5D, which is located on the Y chromosome, contributed to cancer progression in KRAS-mutated tumors. In the same issue of Nature, a team from Cedars-Sinai reported new insights into the consequences of losing the entire Y chromosome.
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