Mission Therapeutics Ltd. has reported clinical trial authorization (CTA) by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for a first-in-human phase I study of MTX-325 for the treatment of Parkinson’s disease. Dosing is expected to commence in the first quarter of next year, with preliminary data to follow later in the year.
Nuclear receptor subfamily 1 group D member (NR1D1), also known as Rev-erbA-α, is highly expressed in the liver, adipose tissue, brain and skeletal muscle.
Seismic Therapeutic Inc. has closed a $121 million series B financing to support progression of the company’s two lead programs through clinical proof of mechanism.
Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy.
Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS).
Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene.
The misassembly of transthyretin (TTR) into toxic amyloid aggregates leads to a variety of degenerative disorders known as TTR amyloidosis (ATTR). Researchers from the Universitat Autònoma de Barcelona and collaborators recently reported on the design and preclinical characterization of PITB, a TTR-selective kinetic stabilizer with potential for the treatment of ATTR.
A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
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