Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).
Sangamo Therapeutics Inc. has received IND clearance from the FDA for ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber neuropathy (iSFN).
Eli Lilly & Co. has elected to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis (ALS) from its collaboration with Verge Analytics Inc. (dba Verge Genomics).
Verge Analytics Inc. (dba Verge Genomics) has prepared and tested new hydrazone and pyrazole 3H-imidazo(4,5-b)pyridine compounds acting as 1-phosphatidylinositol 3-phosphate 5-kinase (PIKfyve) inhibitors.
Scientists at Gachon University and their collaborators investigated the neuroprotective effects of the GPR40 agonist TUG-469 in models of Parkinson’s disease (PD).
While women make up half the world’s population and own two out of every five businesses, there are substantial knowledge gaps about conditions affecting their health – mostly due to decades of research excluding women from clinical trials and investment decisions.
DEAD-box helicase 17 (DDX17) is an RNA helicase involved in the early phases of neuronal differentiation. Researchers have identified a total of 13 patients presenting with neurodevelopmental phenotypes and who harbored de novo monoallelic variants in the DDX17 gene. The phenotype was characterized by intellectual disability, delayed speech and language, as well as motor delay.