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Endocrine/metabolic

VDAC1 inhibitors reported in Abarceo patent

July 17, 2025
Abarceo AB has developed new voltage-dependent anion-selective channel protein 1 (VDAC1) inhibitors reported to be useful for the treatment of diabetes type 1 and 2.
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Cancer

Merck Sharp & Dohme discovers new PTPN1 and PTPN2 inhibitors

July 17, 2025
A recent Merck Sharp & Dohme LLC patent details new substituted isoquinoline derivatives acting as tyrosine-protein phosphatase non-receptor type 1 (PTPN1; PTP-1B) and/or PTPN2 (TCPTP) inhibitors potentially useful for the treatment of cancer.
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Cancer

Childrens Medical Center patents new gasdermin-D activators

July 17, 2025
Work at Childrens Medical Center Corp. has led to the identification of new gasdermin-D (GSDMD) activators reported to be useful for the treatment of cancer.
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3D illustration of tumor
Cancer

PROTAC-mediated DDR1 degradation unlocks tumor immunity

July 17, 2025
No Comments
Discoidin domain receptor tyrosine kinase 1 (DDR1) contributes to tumor progression by promoting the alignment and densification of collagen fibers within the extracellular matrix (ECM), thereby facilitating the development of an immune-excluded tumor microenvironment (TME).
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Diagram of how cells take up glucose from the stomach and insulin from the pancreas
Endocrine/metabolic

An agonist of the BMP receptor to stimulate new β-cell formation in diabetes

July 17, 2025
No Comments
Stimulating the production of β cells in the pancreas of individuals with type 1 diabetes may be a way to renormalize their glucose metabolism. Researchers from Spain and the U.S. may have moved one step closer to this goal by stimulating formation of new β cells in the pancreas of a mouse model of diabetes using the cyclic peptide THR-123, which binds and activates the receptor for bone morphogenetic protein (BMP).
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Biomarkers

Study unveils TNFRSF9 as a marker of abdominal aortic aneurysm

July 17, 2025
No Comments
Abdominal aortic aneurysm is a life-threatening cardiovascular disease characterized by chronic inflammation and irreversible dilatation of the abdominal aorta and is asymptomatic at the early stages of the disease, with diagnosis usually relying on imaging techniques and surgery being the main approach for treating it.
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Vascular system
Cardiovascular

Targeting the long noncoding RNA SRA could counteract myocardial hypertrophy

July 17, 2025
No Comments
Myocardial hypertrophy is a condition characterized by thickening of the ventricular wall and commonly associated with progression to heart failure. It develops when the heart is subjected to biomechanical stress or neurohormonal or hemodynamic stimuli.
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Dollar sign in lightbulb
Respiratory

Cystic Fibrosis Foundation grants new funding to advance development of Prime Medicine’s prime editors

July 17, 2025
No Comments
The Cystic Fibrosis Foundation has agreed to provide Prime Medicine Inc. an additional investment of up to $24 million to continue the development of a gene editing therapy for people with cystic fibrosis. Prime Medicine uses a gene editing technology called prime editing, which enables a wide range of modifications to the DNA with a high degree of precision.
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Neurology/psychiatric

FDA grants orphan drug designation to Amphix Bio’s AMFX-200

July 17, 2025
No Comments
Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor receptor) and ITGB1 (integrin β1) agonist peptide amphiphile scaffold. In preclinical models of acute SCI, a single injection of AMFX-200 into the spinal cord enabled motor neurons from the brain to regrow past the injury site.
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Brain and DNA
Neurology/psychiatric

Voyager Therapeutics adds new gene therapy to Alzheimer’s program

July 17, 2025
No Comments
Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a bifunctional payload.
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