Vesper Bio ApS has disclosed sortilin (NT3; Gp95) antagonists reported to be useful for the treatment of neurodegeneration, cancer, pain, diabetes, diabetic retinopathy, glaucoma, hearing loss and cardiovascular disorders, among others.
Loqus23 Therapeutics Ltd. has closed a £35 million (US$43 million) series A financing, with the aim of supporting its work developing small-molecule somatic expansion inhibitors for the treatment of Huntington’s disease and other triplet repeat disorders.
Italian researchers have presented a case study of pediatric advanced sleep-wake phase syndrome in a 4-year-old girl. Autosomal dominant forms of this syndrome due to loss-of-function mutations in the gene coding casein kinase I isoform delta (CSNK1D) have previously been described.
The E4 variant of the APO gene, the R47H variant of the TREM2 gene, and female sex are three of the strongest risk factors for the development of Alzheimer’s disease (AD). By combining all three of them in a mouse model of tauopathy, researchers at Weill Cornell Medical School have identified microglial inflammation and senescence as processes that occurred more strongly in female mice as tauopathy developed.
MYC is a transcription factor that plays relevant roles in cellular processes such as glycolysis, development, cell differentiation or proliferation. Recent research has associated the transcriptional activity of MYC to the activation of T cells in multiple sclerosis.
After a team at the Hospital Universitari Vall d’Hebron found that histone deacetylase 3 (HDAC3) has a role in the chronic inflammatory response in the central nervous system in models of traumatic brain injury, they investigated its role in autoimmune demyelination.
Researchers from Kyverna Therapeutics Inc. presented preclinical data for KYV-101, a first-in-class, fully human autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy being developed for the treatment of patients with B-cell-driven neurologic autoimmune diseases, including multiple sclerosis and myasthenia gravis.
H. Lundbeck A/S and Iambic Therapeutics Inc. have entered into a strategic research collaboration focused on the discovery of a small-molecule therapeutic for the treatment of migraine. The collaboration will leverage Iambic’s artificial intelligence (AI)-powered drug discovery platform.
Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune demyelinating disorder of the optic nerves and the spinal cord that is linked to the presence of anti-aquaporin (AQP4) autoantibodies. Satralizumab, a humanized anti-IL-6 receptor (IL-6R) antibody, has been approved for the treatment of NMOSD but the effects of IL-6R inhibition in myelitis severity have not been fully explored.
At the 2024 European College of Neuropsychopharmacology (ECNP) Congress, researchers have presented work that could lead to ways to boost brain development and prevent neurodegeneration in individuals with Down syndrome.
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