Brenig Therapeutics Inc. has disclosed leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others.
One of the main issues encountered by therapeutic antibodies for the treatment of neurodegenerative disorders such as Alzheimer’s disease (AD) is the difficulty of crossing the blood-brain barrier (BBB) to access the central nervous system. To overcome these problems, several attempts have been made using endothelial transmembrane proteins where antibodies can bind. One of these efforts involves transferrin receptor 1 (TfR1).
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y, for the treatment of methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS).
Virpax Pharmaceuticals Inc. expects to submit an IND application for Envelta (MET-LENK) around the middle of next year following review of comments made by the FDA on the company’s pre-IND submission. If successful, first-in-human trials would also be expected to begin around the middle of the year.
Researchers led by faculty at the Ohio State University College of Medicine have identified a previously unrecognized connection between microtubule-associated protein 6 (MAP6) and the Kv3.1 voltage-gated potassium (K+) channel – two proteins with different functions. The physical interaction between two neuronal proteins appears to maintain normal movement, anxiety and long-term recognition memory in mice.
Pulkit Grover, associate professor of electrical and computer engineering at Carnegie Mellon University, reported a new approach for electrode placement that explicitly exploits the thresholding phenomenon of neurons to achieve significant gains in focal neural stimulation.
Denali Therapeutics Inc. has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of Alzheimer's disease, atherosclerosis, asthma, nonalcoholic fatty liver disease (NAFLD), multiple sclerosis (MS), experimental autoimmune encephalitis, type 1 diabetes and rheumatoid arthritis, among others.
Aquinnah Pharmaceuticals Inc. has announced preclinical research findings for a novel small-molecule therapeutic designed to slow or stop the progression of Alzheimer's disease and related disorders.