Circio Holding ASA and Neoregen Biotech have commenced a research collaboration on novel circular RNA (circRNA) therapeutics based on Circio’s proprietary Circvec technology. The technology is composed of a modular genetic cassette design for efficient biogenesis of multifunctional circRNAs that can be adapted and applied to multiple purposes.
Sosei Group Corp. and Pharmenable Therapeutics Ltd. have expanded their collaboration to apply their respective technologies to drive novel drug discovery for a second neurological disease target.
The ALDH2 gene encodes mitochondrial aldehyde dehydrogenase 2 (ALDH2), a major acetaldehyde-metabolizing enzyme. Approximately 36% of East Asians (around 560 million people) carry an inactivating E504K missense mutation of the ALDH2 gene, which reduces ALDH2 enzymatic activity. This inactivating mutation has been correlated with several diseases and is strongly associated with type 2 diabetes, body mass index and serum lipids in East Asians.
Curasen Therapeutics Inc. has received a strategic investment of up to $5.8 million from the Alzheimer’s Drug Discovery Foundation (ADDF) to advance CST-3056, an α1A-adrenoceptor (α1A-AR) agonist for the treatment of Alzheimer’s disease.
Researchers from Immunophage Biomedical Co. Ltd. have published details on the discovery and preclinical evaluation of a new CD38 inhibitor being developed for the treatment of mitochondrial myopathy.
Researchers from Heinrich-Heine-Universität Düsseldorf and affiliated organizations presented data from a study that aimed to identify novel neurological biomarkers in distal sensorimotor polyneuropathy (DSPN).
Rejuvenate Bio Inc. has released new preclinical efficacy data for its gene therapy, RJB-0402, in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
Ideaya Biosciences Inc. has announced the selection of a Werner helicase inhibitor development candidate. The candidate is a potent, selective, small-molecule inhibitor of the helicase domain of Werner protein and is being advanced for tumors characterized by high microsatellite instability (MSI-high).
Recurrent driver mutations in FMS-related receptor tyrosine kinase 3 (FLT3) occur in around one-third of patients with de novo acute myeloid leukemia (AML). Although most FLT3 mutations are secondary events in leukemogenesis, they are associated with accelerated clonal expansion and disease progression, and treatment with the tyrosine kinase inhibitor midostaurin has been shown to increase patients’ long-term survival. However, the efficacy of FLT3 inhibitors in eliminating FLT3-mutated clones is variable.
For individuals who develop an unexpected psychosis, there is something to be said for testing them for autoimmune antibodies. And something against. At the 36th Congress of the European College of Neuropsychopharmacology (ECNP) this week, the topic was worth a controversy session, where speakers presented the pros and cons of the approach. Currently used tests have a specificity of 99%. But as Ester Coutinho, consultant neurologist at the University of Coimbra, pointed out, the validity of diagnostic tests depends on the prevalence of the disorder one is looking for as well. Coutinho estimated that autoimmune psychoses account for 1% of psychoses overall.
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