A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS). Read More
The misassembly of transthyretin (TTR) into toxic amyloid aggregates leads to a variety of degenerative disorders known as TTR amyloidosis (ATTR). Researchers from the Universitat Autònoma de Barcelona and collaborators recently reported on the design and preclinical characterization of PITB, a TTR-selective kinetic stabilizer with potential for the treatment of ATTR. Read More
Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene. Read More
Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS). Read More
Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy. Read More
GT Biopharma Inc. has submitted an IND application to the FDA for the development of GTB-3650 for the treatment of patients with CD33+ leukemia. Read More
Seismic Therapeutic Inc. has closed a $121 million series B financing to support progression of the company’s two lead programs through clinical proof of mechanism. Read More
Nuclear receptor subfamily 1 group D member (NR1D1), also known as Rev-erbA-α, is highly expressed in the liver, adipose tissue, brain and skeletal muscle. Read More
A Genentech Inc. patent discloses new benzothiazole compounds acting as von Hippel-Lindau disease tumor suppressor (VHL) inhibitors and reported to be useful for the treatment of ischemia, cancer and anemia. Read More
Merck Sharp & Dohme LLC has patented compounds reported to be useful for the treatment of respiratory syncytial virus (RSV) and metapneumovirus (MPV) infections. Read More
Mission Therapeutics Ltd. has reported clinical trial authorization (CTA) by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for a first-in-human phase I study of MTX-325 for the treatment of Parkinson’s disease. Dosing is expected to commence in the first quarter of next year, with preliminary data to follow later in the year. Read More
Phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors are described in a Relay Therapeutics Inc. patent as potentially useful for the treatment of cancer, particularly, breast and ovarian cancer. Read More
Voronoi and Voronoi Bio Inc. have patented heteroaryl derivative compounds acting as serine/threonine-protein kinase B-raf (BRAF) inhibitors and reported to be useful for the treatment of cancer. Read More
Simcere Pharmaceutical Group Ltd.’s subsidiary Simcere Zaiming Pharmaceutical Co. Ltd. has received FDA clearance of an IND application for SIM-0501, an oral small-molecule inhibitor of ubiquitin-specific peptidase 1 (USP1), for advanced solid tumors. Read More
Are you trying to stay on top of developments in CAR T therapies? Drug resistance? Emerging compounds? Updates from certain regulatory agencies? Want to be the first to learn about new biomarkers or business deals? You can still find these articles in the daily BioWorld e-newsletters or on the website, but if you’re laser-focused on a specific topic, we have a way to highlight the news you need as it happens, whether we’ve published a full-length feature story or a brief update. You asked and we listened. Now, in addition to the daily news lineup, you can create topic alerts to be delivered directly to your inbox or via an RSS reader. It’s easy to set up. Instructions can be found here: https://www.bioworld.com/featured-feeds. Happy holidays! Read More
