Researchers from the Cyprus Institute of Neurology and Genetics and collaborators report the development of a gene silencing therapy for Charcot-Marie-Tooth type 1A.
Merck Sharp & Dohme LLC (MSD) has synthesized new indazole derivatives acting as potassium/sodium hyperpolarization-activated cyclic nucleotide-gated channel 1 (HCN1) and HCN2 blockers potentially useful for the treatment of pain, tinnitus, neurological and mood disorders.
Newco Sononeu Inc. has launched to translate more than a decade of basic research in controlling cells with ultrasound into novel, non-invasive therapies for conditions including peripheral and diabetic neuropathy. The underlying technology of sonogenetics provides the ability to manipulate cellular activity via ultrasound-sensitive ion channels that respond to acoustic pressure. These channels may be endogenous, or could be delivered by targeted vectors to specific cell types.
Accure Therapeutics has reported new oligopeptide derivatives acting as matrix metalloproteinase-2 (MMP-2) and MMP-9 inhibitors. As such, they are described as potentially useful for the treatment of epilepsy, schizophrenia, autism, cancer, mood disorder, sepsis, Alzheimer’s disease and vascular disorders, among others.
Vertex Pharmaceuticals Inc. has patented new macrocyclic sulfonamide orexin OX2 receptor agonists potentially useful for the treatment of amyotrophic lateral sclerosis, obesity, hypertension, retinopathy, multiple sclerosis, narcolepsy, hypersomnia and Parkinson's disease, among others.
Acadia Pharmaceuticals Inc. has reported new G protein-coupled receptor GPR88 agonists that are potentially useful for the treatment of tardive dyskinesia.
Kinase inhibitors have shown success in disease areas such as oncology, but their application in neurodegenerative diseases is still limited. This is mainly due to several challenges, such as the complexity of kinase networks, limited blood-brain barrier permeability and the lack of biomarkers.
Just over a month after emerging from stealth and disclosing a $150 million series A, Korsana Biosciences Inc. is making the leap to the public market via a merger with Cyclerion Therapeutics Inc. The agreement, which is backed by a $370 million private placement from Korsana’s investors, solidly positions the newly merged company as it heads toward the clinic with KRSA-028, a next-generation shuttled antibody targeting amyloid beta for the treatment of Alzheimer’s disease, and builds out a pipeline of neurodegenerative disease candidates.
Researchers from Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have patented new sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers that are potentially useful for the treatment of pain, urinary incontinence, multiple sclerosis, cough and arrhythmia.
Currently available disease management options for Duchenne muscular dystrophy (DMD) are mostly symptomatic. Several strategies based on exon-skipping or gene transfer have been proposed to restore dystrophin expression, but can only address specific subsets of DMD patients and/or provide limited clinical benefits. Upregulating utrophin (UTRN), a structural and functional paralogue of dystrophin, has been proposed as an alternative therapeutic approach that may be suitable for all DMD patients, regardless of their genetic defect.