BioWorld Science - Articles

Biomarkers

De novo variants in DDX39B linked to new neurodevelopmental syndrome

Nov. 7, 2023

The DDX39B gene belongs to the DExD/H-box family of ATP-dependent RNA helicases, playing a vital role in mRNA processing. DDX39B is a component of the TRanscription-EXport (TREX) protein complex, whose pathogenic variants have been recently associated with neurodevelopmental and neurodegenerative disorders.

Neurology/Psychiatric

Takeda Pharmaceutical describes new OX2 receptor agonists for narcolepsy

Nov. 6, 2023

Takeda Pharmaceutical Co. Ltd. has identified orexin OX2 receptor agonists reported to be useful for the treatment of narcolepsy.

Neurology/Psychiatric

Brenig Therapeutics patents new LRRK2 inhibitors for Parkinson’s disease

Nov. 3, 2023

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.

Neurology/Psychiatric

Trontinemab uses a TfR1-binding shuttle module to cross BBB

Nov. 3, 2023

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).

Neurology/Psychiatric

FDA awards orphan drug designation to Huidagene’s CRISPR RNA-editing therapy for MECP2 duplication syndrome

Nov. 2, 2023

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).

Neurology/Psychiatric

Virpax eyes IND filing for Envelta for pain following FDA’s pre-IND comments

Nov. 2, 2023

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.

Neurology/Psychiatric

MAPping protein interactions in schizophrenia reveals functional roles

Nov. 2, 2023

By Subhasree Nag

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.

Neurology/Psychiatric

Denali Therapeutics presents new NLRP3 inflammasome inhibitors

Oct. 31, 2023

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.

Neurology/Psychiatric