Researchers from the Universitat de Barcelona and Oregon Health & Science University have developed a novel gene silencing technique using polypurine reverse Hoogsteen hairpins (PPRH) to target and inhibit the expression of PCSK9.
Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U.
Chinese researchers are preparing the details for the publication of another scientific milestone, the creation of a chimera with a human heart and a kidney developed from human stem cells in pig embryos. These studies aim to address the shortage of immunocompatible organ donors while shedding light on some of the most fundamental questions in developmental biology.
Shanghai Qilu Pharmaceutical Research and Development Centre Ltd. has divulged compounds acting as apolipoprotein A (ApoA; LPA) inhibitors reported to be useful for the treatment of hyperlipidemia, coronary heart disease, ischemic stroke and myocardial infarction.
Doxorubucin is an effective therapeutic in cancer treatment, but it can cause cardiotoxicity and damage the mitochondrial machinery. A new patient-derived and mitochondria-rich human induced pluripotent stem cell derived-cardiomyocyte (hiPSC-CM) model protected by dexrazoxane was developed.
Monte Rosa Therapeutics Inc. has gained IND clearance from the FDA for MRT-8102, a NEK7-directed molecular glue degrader being developed to treat inflammatory conditions linked to NLRP3, IL-1β and IL-6 dysregulation.
Rednvia Co. Ltd. has disclosed alkaline phosphatase tissue-nonspecific isozyme (TNAP) inhibitors reported to be useful for the treatment of ectopic calcification.
Shanghai Circode Biomed Co. Ltd. has obtained IND clearance from the FDA for HM-2002 for ischemic heart disease. It previously received IND clearance in China in January this year.