Voyager Therapeutics Inc. has announced its decision to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Emerging preclinical data indicate the siRNA payload component of VY-9323 does not meet its standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window.

Microglia play a crucial role in neuroinflammation after ischemic brain injury. Previous data have shown that leukocyte immunoglobulin-like receptor B4 (LILRB4) was upregulated in mice after middle cerebral artery occlusion (MCAO), but the role it plays here is not well understood. The role LILRB4 plays in ischemic brain injury was thus investigated. LILRB4 expression was measured in mice at different time points after MCAO.

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.

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.