Loss-of-function mutations in the CTNNB1 gene cause β-catenin deficiency, leading to CTNNB1 syndrome, a rare neurodevelopmental disorder marked by motor and cognitive impairments. Because the disease stems from single-allele mutations that vary widely, a broadly applicable corrective strategy is needed. Since CTNNB1 is dosage-sensitive, therapies must preserve normal regulation, and in some cases may also need to suppress harmful mutant transcripts.

Jazz Pharmaceuticals plc and Saniona AB have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop and commercialize SAN-2355 for epilepsy and other potential indications.

Stimvia s.r.o.’s Uris system, which delivers peroneal electrical transcutaneous neuromodulation, successfully treated the symptoms of Parkinson’s disease. The non-invasive device, originally developed to treat overactive bladder, safely reduced tremors and improved the quality of life of the patients suffering from Parkinson’s.

Receptor-interacting protein kinase 1 (RIPK1) plays a pivotal role in ischemic stroke pathology by mediating necroptosis and promoting neuroinflammation, both of which contribute to secondary brain injury and worsen clinical outcomes.

Medtronic plc posted nearly 50% year-over-year growth in its cardiac ablation business this quarter, with a 72% increase in revenue in the U.S., largely driven by pulsed field ablation. With significant opportunity in renal denervation and a new committee to look at growth including M&A and divestitures and another to review operations, analysts think the company stands a good chance of shedding its “chronic underperformer” image.

G2Gbio Inc. debuted on the Korea Exchange with a ₩52.2 billion (US$37.7 million) IPO Aug. 14. Nearly half of the funds raised will be used to expand Cheongju-si, South Korea-based G2Gbio’s manufacturing plants and help mass produce sustained-release injectable drug products such as GB-5001, its lead candidate in development as a once-monthly formulation of donepezil (Aricept; Eisai Co. Ltd.) for Alzheimer’s disease.

“Our mission is to apply our protein-protein interaction (PPI) big data-generation platform to create novel antibody therapeutics,” Proteina Co. Ltd. CEO Yoon Tae-young recently told BioWorld.