Remove Drugs Remove Gene Expression Remove Protein
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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4.

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AI tool may help doctors select best drugs for COVID patients

pharmaphorum

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest.

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Anti-convulsant drug can modify DNA conformation and interact with chromosome proteins

Scienmag

Brazilian research group shows that valproic acid (VPA), used to treat epilepsy since the 1960s, modulates gene expression in tumor gene models and acts on DNA conformation and the histones in chromatin Results of recent studies involving valproic acid, used for decades as an anti-convulsant drug, show that it can interact with the conformation of (..)

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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

XTalks

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Visium Spatial Gene Expression Solution. Feature barcoding using gel beads. Single Cell Data Analysis.

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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome. Developed with the University of Edinburgh, the EXACT self-contained technology enables MeCP2 protein therapeutic levels while avoiding overexpression-related toxicities.

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EHA 2023: Kura Oncology’s menin inhibitor ziftomenib shows impressive Phase I results

Pharmaceutical Technology

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. GlobalData forecasts total sales from this drug class to exceed $1bn by 2029.

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Discovery suggests new way to target mantle cell lymphoma

Medical Xpress

A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates gene expression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.