New UCD centre targets the biggest bottleneck in rare research
The world’s first translational research center dedicated to urea cycle disorders (UCDs) has been launched in Zürich, marking a significant moment in rare disease innovation.
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Vascularised stem cell organoids advance diabetes therapy
A team of researchers have developed the first vascularised organoid model of human pancreatic islets, which could lead to further development of advanced cell therapies for diabetes.
Nasal spray gene therapy shows potential to treat lung diseases
Researchers at Mass General Brigham have developed a new gene therapy delivery system, AAV.CPP.16, that targets the lungs via nasal spray, which could offer a potential breakthrough in respiratory gene therapy.
New biobank offers hope for sufferers of dry mouth
Mayo Clinic researchers have created the world’s first biobank of human salivary gland tissue-organoids, offering a resource for developing regenerative therapies to treat chronic dry mouth.
New ‘ovaroid’ model research offers hope for reproductive disorders
Researchers have developed the first fully stem cell-derived model of human ovarian organoids, or "ovaroids," offering a powerful new tool for studying infertility, differences in sex development (DSDs), and related reproductive disorders.
New investigational therapy shows promise for treating skeletal disorders
Tyra Biosciences has announced new data showing that its investigational therapy, TYRA-300, improves bone growth and corrects skeletal abnormalities in preclinical models of achondroplasia and hypochondroplasia.
ELRIG’s Drug Discovery 2025 announces event speakers
ELRIG has announced the keynote speakers for Drug Discovery 2025, its flagship life sciences event taking place in Liverpool, England this October.
Targeting PLA2G15 to treat neurometabolic disorders
Scenic Biotech and Stanford University have published a study identifying PLA2G15 as a new drug target in neurodegenerative and metabolic diseases, potentially enabling future treatments for conditions like Niemann-Pick type C.
K Navigator: an AI co-pilot transforming biomedical research
K Navigator, a new AI-powered research co-pilot, is set to transform biomedical science by helping researchers explore complex data and accelerate discoveries.
Targeting the clock: new drug disrupts glioblastoma stem cells
Scientists have developed a new drug, SHP1705, that targets hijacked circadian clock proteins used by glioblastoma stem cells to grow and resist treatment.
New data: Visugromab targets ADC resistance mechanism
CatalYm announces new preclinical data showing that its clinical-stage antibody, visugromab, can overcome resistance to antibody-drug conjugates (ADCs), showing potential to restore anti-tumour immune responses in solid tumours.
Enedra secures funding to advance AI-driven cancer platform
Enedra Therapeutics has secured new funding to advance its AI-driven CASPAROV platform, aimed at developing therapies for difficult-to-treat cancers
High-throughput NaV1.9 assay to advance pain therapies
Metrion Biosciences has introduced a breakthrough NaV1.9 screening assay, aimed at overcoming historical challenges in pain research and advancing the development of non-opioid treatments.
New enzyme tech offers hope for mitochondrial disease treatment
Japanese researchers have developed a new enzyme technology that can precisely alter the levels of mutated mitochondrial DNA in patient-derived stem cells, offering a promising new approach for treating mitochondrial disorders.
Eli Lilly backs Creyon’s quantum chemistry platform in $1B RNA drug deal
The partnership allows for the further development of AI-designed RNA-targeted therapies for a range of diseases.
