Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).
Researchers from Ashvattha Therapeutics LLC recently reported on ASH-41020, a new dendranib compound acting as a CSF-1R inhibitor with anti-inflammatory and immunomodulatory effects.
Monoacylglycerol lipase (MAGL), a member of the serine hydrolase family expressed in the brain and peripheral tissue, is a key enzyme in the hydrolysis of monoglycerides, converting 2-arachidonoyl glycerol (2-AG) into arachidonic acid and glycerol. MAGL inhibition has been previously shown to induce anxiolytic and analgesic phenotypes in animal models. Researchers from Janssen Pharmaceutica NV recently reported the discovery of novel noncovalent MAGL inhibitors.
A Spanish study led by scientists from the Cajal Institute and the National Center of Oncological Research (CNIO) combined the power of artificial neural networks and biological neuronal circuits to identify abnormal brain activity produced by secondary metastases in the CNS and classify these tumors. The work, published online on Aug. 30, 2023, in Cancer Cell, showed how damage in the brain did not depend on the tumor size but on the effect it produced on neuronal circuits, interrupting cell communication.
A DE Shaw Research LLC patent reports N3-substituted uracil compounds acting as transient receptor potential cation channel subfamily A member 1 (TRPA1) antagonists. As such, they are reported to be useful for the treatment of pain, fibrosis, autoimmune disease, neurological, dermatological, cardiovascular, respiratory and gastrointestinal disorders, among others.
CCM Biosciences Inc. has launched with a focus on discovering and developing novel drugs, including small molecules, gene therapies, biologics and nanomedicines, and companion diagnostic tests.
Research at Vanderbilt University has led to the identification of new muscarinic M5 receptor antagonists reported to be useful for the treatment of psychiatric disorders.
“I am not a fortune teller, nor am I a gambler. I will make no bets,” Lorraine Kalia told the audience at the 2023 International Congress of Parkinson’s Disease and Movement Disorders. “But I am optimistic.” At the meeting, which is being held in Copenhagen this week, Kalia, who is a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, was giving an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”
Eikonizo Therapeutics Inc. has described histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of cancer, multiple sclerosis, systemic lupus erythematosus, neurodegeneration, obesity, Alzheimer’s disease, Parkinson’s disease and Huntington’s disease, among others.