Scripps Research Institute and Vova Ida Therapeutics have divulged novel compounds that may inhibit NAD consumption and/or increase NAD synthesis, reported to be useful for neurodegenerative, degenerative and metabolic disorders, including amyotrophic lateral sclerosis, diabetes, nonalcoholic fatty liver disease (NAFLD), Parkinson's disease and renal, metabolic and retinal diseases, among other disorders.
As the resident innate immune cells of the brain, microglia are emerging as key drivers of neurological diseases, but as yet there is no systematic way of exploring their potential as drug targets.
Pasithea Therapeutics announced positive results from a preclinical proof-of-concept study of PAS-002, the company's DNA tolerizing vaccine construct encoding GlialCAM, a molecule that has been recently identified in the brain's white matter, which is the portion of the brain attacked in multiple sclerosis (MS).
An inhibitor of sodium/glucose cotransporter 2 (SGLT2), luseogliflozin, was found to have protective effect in a model of acute ischemic stroke in research by Kyushu University scientists and their collaborators.
The therapeutic validity of RNA editing tools in vivo remains largely unknown in disease models both in terms of RNA editing efficacy and therapeutic improvement of disease-related symptoms. In recent work, scientists from the Institute of Neuroscience at the Chinese Academy of Sciences and colleagues evaluated RNA correction therapy in a mouse model that recapitulates the phenotype of human dominant-inherited deafness.
DexTech Medical announced that its application for a phase I clinical trial of OsteoDex in patients with multiple myeloma has been submitted to the Medical Products Agency on June 23, 2022 and it has been approved and granted permission August 10, 2022.
Aro Biotherapeutics announced that the FDA has granted orphan drug designation for ABX-1100, its investigational Centyrin-siRNA conjugate, for the treatment of Pompe disease.
Cure Rare Disease (CRD) announced that the FDA has approved the first-in-human IND for CRD-TMH-001, its first therapeutic candidate using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy.
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