Many patients who survive myocardial infarction go on to experience heart failure. Researchers at Qilu Hospital of Shandong University found that bone morphogenetic protein 1 (BMP1) was upregulated in patients after myocardial infarction, as well as in a mouse model of the same condition.
The FDA has granted orphan drug designation to FRF-001, the FOXG1 Research Foundation’s lead gene therapy candidate for the treatment of FOXG1 syndrome. This follows the FDA’s earlier award of rare pediatric disease designation to the investigational therapy.
CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China with SYH-2070 injection, a double-stranded small interfering RNA (siRNA) drug for the treatment of hypertriglyceridemia or mixed hyperlipidemia.
Convelo Therapeutics Inc. has presented data on their 3-β-hydroxysteroid-Δ8,Δ7-isomerase (EBP) inhibitor CVL-1001 as a remyelinating compound for treating multiple sclerosis.
In pulmonary hypertension (PH), upregulated signaling by ActRIIA ligands, such as myostatin, activin A and GDF11, activates SMAD2/3 mediated pathways that drive pulmonary vascular remodeling and right ventricular dysfunction. Therapeutic blockade of these ligands using ligand traps such as sotatercept improves vascular remodeling and ameliorates PH pathology.
Generating gametes from nonreproductive tissues could help overcome infertility. Previous studies have successfully transformed stem cells into viable oocytes through cellular reprogramming. Scientists at Oregon Health & Science University (OHSU) developed a method to derive them from skin cells via somatic cell nuclear transfer (SCNT), unlocking a mechanism that blends mitosis and meiosis. Now, the researchers have taken another step forward by generating fertilizable eggs from human skin cells.
Antengene Corp. Ltd. and Antengene Discovery Ltd. have divulged phospholipid hydroperoxide glutathione peroxidase, mitochondrial (PHGPx; GPX4) inhibitors acting as ferroptosis inducers reported to be useful for the treatment of cancer.
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