When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc.
Recent evidence suggests the gastrin/cholecystokinin-2 receptor (CCK2R) is a potential target for radiolabeled theranostics in cancer, but the stability of minigastrin analogues remains unresolved. Researchers from the National University of Singapore aimed to develop a peptide with improved tumor uptake and prolonged retention in CCK2R+ cancers.
At the 2025 European College of Neuropsychopharmacology (ECNP) Congress in Amsterdam this week, researchers presented new findings on the role of endocannabinoids in stress, addiction … and the integrity of the blood-brain-barrier.
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
Angelman syndrome is a rare genetic, nondegenerative and neurodevelopmental disorder caused by mutations affecting the expression of maternal UBE3A, which is expressed in neurons and is a key protein for neuronal morphology and correct synaptic functioning. The disease is characterized by intellectual disability, defects in movement and sleep disruption, among others.
Parkinson’s disease (PD) is a progressive disease characterized by loss of dopaminergic neurons in the substantia nigra, which lead to motor symptoms. Aromatic-L-amino-acid decarboxylase (AADC) converts levodopa into dopamine and glial cell line-derived neurotrophic factor (GDNF) promotes the survival, growth and regeneration of dopaminergic neurons. VGN-R09b is an adeno-associated viral vector (AAV)-based AADC and GDNF combination gene therapy that is delivered to the striatum for the treatment of PD.
Primary sclerosing cholangitis is an autoimmune disease affecting the liver in which there is reduced expression and function of the bile acid receptor GPBAR1 in the cholangiocytes.
Recurv Pharma Inc. has developed a novel taxoid compound formulated in an oil-in-water nanoemulsion, named RP-001, as a potential therapeutic approach for the management of pancreatic ductal adenocarcinoma (PDAC), either as a single agent or combined with immune checkpoint inhibitors.
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
Japan’s investor community is ramping up both inbound and outbound investment to create a cross-border fertile hub of innovation in Japan, investors said during a panel discussion at Bio Japan 2025 held in Yokohama Oct. 8 to 10.
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