Aeovian Pharmaceuticals Inc. has prepared tricyclic fused imidazole compounds acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors reported to be useful for the treatment of neurodegeneration, inflammation, fibrosis, systemic scleroderma and more.
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.
Neurogene Inc.’s stock sank 43% on news that its phase I/II gene therapy, NGN-401, resulted in a serious adverse event in a pediatric Rett syndrome patient receiving the highest dose.
Scientists at Gachon University and their collaborators investigated the neuroprotective effects of the GPR40 agonist TUG-469 in models of Parkinson’s disease (PD).
The EMA has changed its mind about an earlier decision that the risks of Leqembi (lecanemab) outweigh the benefits and is now recommending the Alzheimer’s disease drug is approved for a subgroup of patients. That follows an appeal by Eisai Co. Ltd. and a re-examination of the data, after details relating to 274 patients with two copies of the ApoE4 gene were removed from the file.