Researchers from the Centre for Addiction and Mental Health (CAMH) and affiliated organizations presented preclinical data for the α5-GABA-A receptor (α5-GABAAR) positive allosteric modulator GL-II-73 (DPX-101), which is being developed by Damona Pharmaceuticals Inc. for the treatment of Alzheimer’s disease and other cognitive and neurological disorders.
The Brigham and Women's Hospital Inc., Massachusetts General Hospital and UCL Business Ltd. have jointly patented new benzo[c][l,2,5]thiadiazolyl compounds and radiolabeled derivatives targeting α-synuclein, amyloid-β (Aβ) protein and microtubule-associated protein tau.
Early dysfunction of the endothelial/blood-brain barrier (BBB) is involved in the pathogenesis of cerebral small vessel disease (SVD), which is a contributor to about 50% of dementias. Sphingosine-1-phosphate (S1P) is a sphingolipid that regulates the BBB integrity when bound to its receptor S1P1 on endothelial cells.
Cure Rare Disease has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3).
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with progressive muscle degeneration. There is currently no approved treatment targeting the underlying cause of the disease.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Researchers from Massachusetts General Hospital and affiliated organizations have published preclinical data for [18F]CNL-02, a positron emission tomography (PET) radioligand targeting mitochondrial complex I (MC-I) that is being developed for the diagnosis of Alzheimer’s disease and other neurodegenerative disorders.
Researchers from Nanjing Drum Tower Hospital reported findings from their evaluation of the role of NLR family CARD domain containing 5 (NLRC5) in post-stroke neuroinflammation. Immunofluorescence staining of mouse brains revealed that NLRC5 was mainly expressed in microglia.
Navega Therapeutics Inc. has received a $4 million Translational Science grant from the California Institute for Regenerative Medicine (CIRM) to support its work addressing neuropathic pain. The grant will fund the final preclinical development studies of NT-Z001 leading to an IND submission.