NIH Director's Blog: Drug Discovery

MARCH 8, 2016

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain.

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DNA Drugs Small Molecule FDA Approval 52

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. “The first printout of the human genome to be presented as a series of books, displayed in the 'Medicine Now' room at the Wellcome Collection, London. It fills up an entire bookcase ! No longer.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. “The first printout of the human genome to be presented as a series of books, displayed in the 'Medicine Now' room at the Wellcome Collection, London. It fills up an entire bookcase ! No longer.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

FEBRUARY 13, 2024

Some algorithms simply look for important similarities between phage and bacterial DNA sequences, a signal of the extensive gene-swapping that goes on when a particular virus and bacteria have shared history. Middle and Right) Once a bacteriophage lands on a cell, it injects its DNA through a tube (purple) into the unwilling host.

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Therapies DNA Virus Clinical Trials 118

Agency IQ

APRIL 12, 2024

Additionally, six of these comments came from the same organization (Integrated DNA Technologies). Thus, they also provided an opportunity for the FDA to gauge which aspects of CGT product development presented obstacles, where common points of confusion lie, and where guidance would be most useful in addressing these concerns.

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Production FDA Therapies Biochemical Assays 40

Drug Target Review

SEPTEMBER 10, 2024

To achieve an aggressive timeline from DNA to investigational new drug (IND) application, drug developers have strengthened collaborations with contract development & manufacturing organisations (CDMOs) to expedite drug development. The picture is far more complex.

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Drug Development Clinical Supply Production Protein Expression 65

Codon

MAY 30, 2023

This week: A way to measure a transgene’s expression in the brain using ultrasound, a DNA sequencing method that uses 1000x less reagents, and base editors get even smaller. An engineered version of this protein can convert DNA bases with efficiencies up to 92%. so this Digest will be published more irregularly.

DNA 52

DNA RNA Engineering Vaccine 52

Codon

MAY 30, 2023

This week: A way to measure a transgene’s expression in the brain using ultrasound, a DNA sequencing method that uses 1000x less reagents, and base editors get even smaller. An engineered version of this protein can convert DNA bases with efficiencies up to 92%. so this Digest will be published more irregularly.

DNA 52

DNA RNA Engineering Vaccine 52

The Pharma Data

OCTOBER 14, 2020

Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakht’s study. The study led by Dr. Sergei Rudchenko, Ph.D., 1 Noyce RS, et al. 2018) PLoS One. 13(1):e0188453.

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Vaccine Pharmaceuticals Small Molecule Virus 40

Agency IQ

MARCH 29, 2024

Such a review will assess whether the use may present unreasonable risk to health or the environment and ensure that EPA can prevent future unsafe environmental releases of the chemical substances subject to this SNUR.

Regulations 40

Regulations Compliance Production Government 40

Agency IQ

MAY 31, 2024

Specifically , the sponsor is proposing an indication for the detection of “colorectal cancer derived alterations in cell-free DNA from blood collected in the Guardant Blood Collection Kit.” The Shield blood test is an in vitro diagnostic (IVD) product intended to screen for colorectal cancer (CRC) markers in blood.

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FDA Laboratories Compliance Packaging 40

Agency IQ

AUGUST 11, 2023

EMA’s investigation ended up revealing even more causes of nitrosamine impurities in drugs, discovering, for example , that formation happened during the heat-sealing process of in certain products using blister-packaging. Exposure to high heat during the storage process could also cause nitrosamines to form, according to the investigation.

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FDA Science Production Regulations 40

Codon

APRIL 2, 2024

in physics at Princeton University, he had a remarkable idea: What if it were possible to build a circuit out of DNA, rather than electronics, and use it to “program” a living cell? Slides from a lab talk presented in Heidelberg, Germany in 1998. In 1997, as Michael Elowitz was studying for a Ph.D.

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RNA Engineering Molecular Biology Synthetic Chemistry 56

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). It aims to correct the faulty instructions at the DNA level, straightforward, at least conceptually, because mutations are deletions, of part or all of the gene. That’s not the case for ALS.

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Therapies FDA Clinical Trials DNA 111

The Pharma Data

JANUARY 21, 2021

CABENUVA, a co-packaged kit with two injectable medicines, offers people living with HIV a new approach for maintaining viral suppression. Results from these trials were presented at the 2019 Conference on Retroviruses and Opportunistic Infections (CROI). Global Head, Janssen Research & Development, Johnson & Johnson.

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FDA Approval Treatment RNA FDA 52

Codon

JUNE 26, 2023

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. Anyone who has tried to engineer a cell knows how tedious it can be.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. Anyone who has tried to engineer a cell knows how tedious it can be.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

APRIL 23, 2023

This newsletter’s format has shifted again, as I try to figure out the most enticing way to present this stuff. Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Please send me your feedback.

Vaccine 52

Vaccine DNA Virus Engineering 52

Codon

APRIL 23, 2023

This newsletter’s format has shifted again, as I try to figure out the most enticing way to present this stuff. Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Please send me your feedback.

Vaccine 52

Vaccine DNA Virus Engineering 52