Newco Vesalic Ltd. has formed to take forward research indicating extracellular vesicles secreted by skeletal muscle cells carry toxic payloads that are key drivers of motor neuron diseases, including amyotrophic lateral sclerosis. The discovery of this process, which is largely external to the brain and the central nervous system, has opened up new targeting possibilities, and Vesalic is now working on in vivo studies to demonstrate preclinical proof of concept.
Positive results from the second of two phase III trials set Compass Pathways plc’s synthetic psilocybin treatment, COMP-360, on track for a potential U.S. FDA approval within a year as the first classic psychedelic cleared for treatment-resistant depression.
The variety of organoids that can be developed in vitro is enabling major advances. Depending on the type of tissues and the research goals, these small 3D cell-based structures that mimic real tissue offer certain advantages over animal models. Scientists at the University of Padova in Italy have created human neuromuscular organoids to reproduce cancer-induced muscle cachexia, a condition that murine models do not accurately replicate.
Hunter syndrome, also called mucopolysaccharidosis II, is an X-linked genetic lysosomal disorder caused by loss-of-function mutations in the IDS gene, encoding iduronate-2-sulfatase (I2S). I2S is a lysosomal enzyme responsible for the cleavage of glycosaminoglycans (GAGs), and its deficiency results in accumulation of GAGs leading to a multisystemic disorder.
Although activation of the μ-opioid receptor (MOR) provides potent analgesia and remains central to pain management, its agonists are associated with adverse effects such as respiratory depression, tolerance, dependence and pruritus. Bifunctional μ-/κ-opioid receptor (MOR/KOR) agonists aim to retain MOR-driven analgesia while leveraging the antipruritic and complementary analgesic effects of KOR activation to improve overall therapeutic tolerability.
Researchers have explored the effects of human umbilical cord mesenchymal stem cells (hUC-MSCs) on cognitive recovery in anti-N-methyl-D-aspartate receptor (NMDAR) encephalitis, a rare, neuronal antibody-mediated disorder characterized by neuropsychiatric symptoms and impaired cognitive function. hUC-MSCs have previously shown immunomodulatory and anti-inflammatory properties.
A circuit formed by tumor, immune and nervous systems triggers cancer cachexia and anorexia, the excessive loss of weight, muscle and fat experienced in some cancer types. A new study is the first showing these three actors of a triangle interaction that initiates and feeds the process.
Nanjing Anji Biotechnology Co. Ltd. has discovered 2-oxoglutarate dehydrogenase, mitochondrial (OGDH; OGDC-E1) inhibitors. They are reported to be useful for the treatment of schizophrenia, liver fibrosis, liver cancer, diabetic nephropathy, heart failure, Alzheimer’s, Parkinson’s and Huntington’s disease, among others.
Vascular cognitive impairment and dementia (VCID) and cerebral small vessel disease are among the leading causes of dementia, where inflammation is known to play a crucial role. Researchers from Augusta University in the U.S. hypothesized that inhibiting the NLRP3 inflammasome with MCC-950 may improve cognitive decline and cerebral blood flow in a murine model of VCID.
Nxera Pharma Co. Ltd. has announced the achievement of an early development milestone by Centessa Pharmaceuticals plc for ORX-489, triggering a payment to Nxera under the companies’ research collaboration.
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