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BioWorld - Thursday, January 8, 2026
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Infection

MRX-5 to treat Mycobacterium abscessus infections

May 9, 2024
New treatment options for treating Mycobaterium abscessus infections are needed. Previous findings had identified the leucyl-tRNA synthetase inhibitor MRX-6038 to have significant activity against M. abscessus. The aim of this new study was to focus on the activity of MRX-5, the oral prodrug of MRX-6038, both in vivo and in vitro.
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Concept art for prenatal genetic testing and whole genome sequencing.
Genetic/congenital

ASGCT: In utero interventions can prevent organ damage after birth

May 9, 2024
By Mar de Miguel
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
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T cells attacking cancer cells
Immuno-oncology

HRYZ-T102, mutated AFP-specific TCR engineered T-cell therapy with improved functionality

May 8, 2024
α-Fetoprotein (AFP) is a tumor-associated antigen and an ideal target for T-cell receptor T-cell (TCR-T) therapy. While the safety of AFP-targeting TCR-T products has been previously demonstrated in early clinical trials, the efficacy of these cell therapies is still modest, warranting further research.
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Ocular

CREB-activating P1-CPP induces retinal ganglion cell survival in glaucoma model

May 8, 2024
Previous findings had shown that injecting pepatin-1 prevented the death of retinal ganglion cells (RGCs) in rats with ocular hypertension. Additionally, transcriptomic analysis in RGCs revealed cAMP response element-binding protein (CREB) signaling to be activated by peptain-1 conjugated with a cell-penetrating peptide, named P1-CPP.
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Lab mouse
Immuno-oncology

New mouse model for preclinical research in human IL-12 therapy

May 7, 2024
Researchers from Gempharmatech Co. Ltd. presented a new humanized mouse model for preclinical research into the functionality and therapeutic potential of IL-12.
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3D illustration demonstrating antibody-drug conjugate.
Immuno-oncology

GB01-VA-PL2202, a Claudin-6-targeting ADC with potent antitumor efficacy and bystander killing activity

May 7, 2024
Researchers from ADC Therapeutics SA presented the discovery and preclinical evaluation of a novel camptothecin-based Claudin-6-specific antibody-drug conjugate (ADC), GB01-VA-PL2202.
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Biomarkers

miR-199a-3p and miR-100-5 have protective role in autoimmune hepatitis

May 7, 2024
MicroRNAs (miRNAs) are small noncoding RNAs that play important roles in immune response regulation in autoimmune disease such as autoimmune hepatitis. Researchers investigated whether Δ8-tetrahydrocannabinol (THC), which is present in marijuana, could prevent the development of autoimmune hepatitis in treated mice by altering miRNA expression.
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3D pancreas illustration
Cancer

CCR6 as therapeutic target in pancreatitis and PDAC

May 7, 2024
Pancreatic ductal adenocarcinoma (PDAC) has a poor prognosis, in part due to the immunosuppression surrounding the tumor mediated by regulatory T cells (Tregs), thus preventing effective responses to immune therapy.
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3D rendering conceptualizing theranostics
Cancer

[64Cu]PD-32766 and [177Lu]PD-32766, a new theranostic pair for CAIX-expressing tumors

May 6, 2024
Researchers from Peptidream Inc. presented preclinical characterization of novel carbonic anhydrase IX (CAIX)-targeting radiopeptides, [64Cu]PD-32766 and [177Lu]PD-32766, being developed for the diagnosis and treatment of clear cell renal cell carcinoma (ccRCC), respectively.
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Pseudomonas aeruginosa
Infection

Activity and mechanism of action of antibiotic potentiator JS-238

May 6, 2024
There is a compelling need to produce new antibiotics that hamper severe opportunistic infections, especially in immunocompromised individuals, such as those caused by gram-negative Pseudomonas aeruginosa.
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