In Duchenne muscular dystrophy (DMD), deficiency of dystrophin leads to cardiomyocyte membrane instability, abnormal calcium influx, and progressive fibrotic remodeling of cardiac tissue. Histone deacetylase 6 (HDAC6) contributes to disease progression by regulating cytoskeletal dynamics and proteostasis in dystrophic muscle cells. Consequently, inhibition of HDAC6 represents a potential therapeutic strategy for addressing both the skeletal and cardiac manifestations of DMD.
Entering its first major cardiovascular disease collaboration with a biopharma company, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals Inc. to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to South San Francisco-based Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes.
Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have disclosed amide-substituted cycloalkyl compounds acting as relaxin receptor 1 (RXFP1; LGR7) agonists potentially useful for the treatment of heart failure and hypertension.
Targeting rare cardiomyopathy conditions with no approved disease-modifying therapies, Atrium Therapeutics Inc. started operations in San Diego, assuming the public listing and technology once held by Avidity Biosciences Inc., an RNA company acquired by Novartis AG for about $12 billion. Novartis closed the M&A, announced last October, as Atrium unveiled its pipeline and a $270 million cash position.
Retex Pharmaceuticals Inc. has patented vasopressin V2 receptor antagonists potentially useful for the treatment of hyponatremia, cardiovascular disorders, autosomal dominant and autosomal recessive polycystic kidney.
Hepatic ischemia-reperfusion injury (HIRI) is a severe pathologic condition associated with poor outcomes when individuals suffer from hemorrhagic shock, liver resection or transplantation surgery, leading to severe liver impairment and sometimes dysfunction in other organs. Chinese researchers have explored the potential of prolyl 3-hydroxylase OGFOD1 as a target for HIRI management, since it has been reported as a crucial regulator of gene expression, especially for translation.
In previous work, researchers from CEINGE (Naples, Italy) and collaborating institutions developed an alternative gene therapy strategy based on the expression of a secreted fusion protein, including the extracellular portion of the human LDLR for LDL binding, linked to rabbit transferrin (TF), targeting the transferrin receptor (TFr), resulting in internalization and uptake of LDL particles. They have now published more recent work to further improve the efficacy and safety of this strategy.
The potential of G9A as a therapeutic target was investigated in vitro in vascular smooth muscle cells as well as in vivo in a murine model of vascular intimal hyperplasia.
Animate Biosciences Inc.’s lead therapeutic peptide MBb32 produced a significant reduction in cardiac scar tissue and subsequent recovery of heart function in a mouse model of severe myocardial injury.
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