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3D illustration of interferon-alpha molecular structure
Infection

IFNα-like small molecules successfully inhibit HBV replication

Nov. 15, 2023
It’s known that interferon-alpha (IFNα) activates interferon-stimulated genes (ISGs) and disrupts the hepatitis B virus (HBV) replication cycle. Pegylated (PEG)-IFNα has been widely used for its immunomodulatory and antiviral properties but it is not always well tolerated and thus its use is limited.
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Streptococcus pneumoniae in lungs
Infection

Collaboration evaluates COMBINE’s in vivo pneumonia model

Nov. 15, 2023
The Innovative Medicines Initiative’s COMBINE (COllaboration for prevention and treatment of MDR Bacterial INfEctions) project is developing a standardized in vivo pneumonia model to test small-molecule antibiotics.
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Brain and DNA
Genetic/Congenital

POLD3 variant linked to immunodeficiency, neurodevelopmental delay

Nov. 15, 2023
Inborn errors of immunity comprise a group of several diseases, the most severe of which are immunodeficiency disorders. The latter are characterized by defective T-cell functioning leading to impaired immunity.
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Neurology/Psychiatric

Satellos reveals drug target, lead candidate in Duchenne program

Nov. 15, 2023
Satellos Bioscience Inc. has disclosed further information on its lead drug program in Duchenne muscular dystrophy.
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Art concept for gene therapy research
Neurology/Psychiatric

Solid Biosciences gains IND clearance for SGT-003 gene therapy in Duchenne

Nov. 15, 2023
Solid Biosciences Inc. has received FDA clearance of its IND application for SGT-003, the company’s next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD). The planned first-in-human phase I/II trial will enroll pediatric patients with DMD to receive SGT-003 as a one-time intravenous infusion.
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AI generated illustration of lungs in the human body
Respiratory

Characterization of NBD1 stabilizers for cystic fibrosis

Nov. 15, 2023
ΔF508-CFTR is the most common CFTR mutation in cystic fibrosis (CF), which leads to destabilization of CFTR’s first nucleotide-binding domain (NBD1), contributing centrally to defective ΔF508-CFTR folding, trafficking and function.
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Natural killer cell attacking cancer cell
Immuno-oncology

SNK-02 cell therapy clears tumors in HER2-driven tumor models

Nov. 15, 2023
Researchers from Nkgen Biotech Inc. have developed an anti-HER2 CAR NK cell therapy, named SNK-02, as a potential immunotherapeutic for HER2-overexpressing tumors. SNK-02 was tested in a xenografted murine model of cancer.
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Cancer

ADI-925 is potential universal cell therapy in cancer

Nov. 15, 2023
Researchers from Adicet Bio Inc. reported on the preclinical development of ADI-925, an engineered Chimeric Adapter (CAd) γδ1 T-cell therapy that targets major histocompatibility complex class I chain-related protein A/B (MICA/MICB) and ULBP1-6 expressed in tumor cells.
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Mitochondria illustration
Endocrine/Metabolic

Mitophagy inducer prolongs life and prevents neurodegeneration

Nov. 15, 2023
By Mar de Miguel
A new derivative of coumarin, a natural plant product abundant in cinnamon, could hold the key to healthy aging. Researchers at the Buck Institute have shown that it extended life span and prevented neurodegenerative disease in worms and mice. The drug, a TFEB gene inducer called MIC, promoted mitochondria recycling (mitophagy) but also interacted with lysosomes, which could have multiple applications. The scientists published the results of this aging and mitophagy study on Nov. 13, 2023, in Nature Aging.
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Antibodies
Neurology/Psychiatric

Vectory Therapeutics closes series A financing to advance vectorized antibody into clinic

Nov. 14, 2023
Vectory Therapeutics BV has closed a €129 million ($138 million) series A financing to advance its vectorized antibody programs in neurodegenerative diseases.
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