Iecure Inc. has received clearance from the Australian Therapeutic Goods Administration (TGA) of the company’s clinical trial notification (CTN) for ECUR-506 (formerly GTP-506), an investigational therapy in development for the treatment of ornithine transcarbamylase (OTC) deficiency in pediatric (or neonatal) patients.
Korro Bio Inc. has nominated its first development candidate, KRRO-110, for the potential treatment of α1-antitrypsin deficiency. KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant.
Researchers from Mitsubishi Tanabe Pharma Corp. and affiliated organizations have described the discovery and preclinical evaluation of a novel adiponectin receptor (AdipoR)-activating monoclonal antibody, named AdipoRaMab, being developed for the treatment of type 2 diabetes and nonalcoholic steatohepatitis (NASH).
Rhythm Pharmaceuticals Inc. has presented preclinical data on its new drug candidate, RM-718, a next-generation melanocortin MC4 receptor (MC4R) agonist for weekly administration with a potential development path for hypothalamic obesity.
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.
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.
CSPC Pharmaceutical Group Ltd.’s SYH-2053 injection has obtained clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials in China.
REGN-4461 (mibavademab) is a monoclonal antibody targeting leptin receptor (LEP-R) under development by Regeneron Pharmaceuticals Inc. It acts as an allosteric agonist and does not compete with leptin binding to LEP-R.
A laboratory technique used to generate pluripotent stem cells from any tissue, cellular reprogramming, has led a group of researchers to the discovery of a process that could have an impact on natural tissue repair.