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Target-directed cancer: protein-ligand interactions  

Drug Target Review

The mission of the CCDD is to discover novel small-molecule therapeutics for the treatment of cancer and progress them to hypothesis testing phase 1 clinical trials. We support the drug discovery projects of the CCDD with assay development and screening, biophysics, structural biology and recombinant protein production.

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Eloxx Pharmaceuticals to Report Third Quarter 2020 Financial Results and Provide Business Update on November 5, 2020

The Pharma Data

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

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Targeting a human protein may stop Ebola virus in its tracks

The Pharma Data

Unfortunately, in a Phase 3 clinical trial, treatment with remdesivir did not make a difference in mortality for Ebola virus disease patients. Without the stop sign, viral transcription no longer follows the correct order, which has a negative impact on viral protein production,” says Fang.

Virus 52
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A Look Back

Dark Matter Blog

At Biogen, my group’s work covered a range of technology development areas including protein production in mammalian cells, intracellular protein delivery (our work on HIV Tat started the field of cell-penetrating peptides) and novel gene delivery vehicles, as well as biological drug discovery and development.

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Proteogenomics reveals markers of chemotherapy resistance and outcome in triple negative breast cancer

Broad Institute

LIG1 ’s protein product is a critical component of lagging strand DNA synthesis that connects Okazaki fragments (small DNA segments that must be connected to complete the synthesis reaction). Support for this study was provided by the National Cancer Institute.

DNA 52
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Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

Not only are assays useful tools for characterizing products early in development; they can also be used to set “benchmarks,” allowing sponsors to verify that the lots of the product used during clinical trials and eventually, for commercial distribution, continue to adhere to the same standards specified in the original product’s design.

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Stoke Therapeutics Announces Presentations Related to the Company’s Work to Advance STK-001, the First Potential New Medicine to Target the Underlying Cause of Dravet Syndrome at the American Epilepsy Society (AES) 2020 Annual Meeting

The Pharma Data

Clinical Trials. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. December 6, 2020 12:00 PM – 1:30 PM; Track: 7. Antiepileptic Drugs / 7B. About STK-001.