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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The Premier Consulting Blog

MARCH 7, 2024

The analytical package, consisting of release, stability, and characterization tests, includes data generated throughout the product development and manufacturing process. For gene therapy vectors, this testing encompasses production cell lines, master and working cell banks (MCB, WCB), and virus banks.

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Broad Institute

MAY 14, 2025

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells. Like Cas9, IscB enzymes cut DNA at sites specified by an RNA guide.

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Drug Target Review

DECEMBER 5, 2024

2 Structure and function of AAV capsids in gene therapy Wild-type AAV is composed of a protein shell (capsid) that contains a single-stranded DNA genome encoding proteins involved in viral replication, structure and assembly. Adeno-associated virus as a delivery vector for gene therapy of human diseases. Labcompare. 2024;9(1):78.

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The Pharma Data

OCTOBER 19, 2020

Spectrum’s self-contained saliva collection kit provides critical sample consistency while suspending and neutralizing viral RNA transcripts completely inactivating the live virus. ABOUT SPECTRUM SOLUTIONS AND SPECTRUM DNA. This press release features multimedia. EUA validated viral inactivation data.

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The Pharma Data

OCTOBER 14, 2020

The research is part of an ongoing collaboration between Columbia University and Tonix that focuses on T cell and antibody responses to SARS-CoV-2 (CoV-2), the virus that causes COVID-19. The research encompasses two projects. The study led by Dr. Ilya Trakht, Ph.D., The study led by Dr. Sergei Rudchenko, Ph.D., About TNX-1800.

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The Pharma Data

SEPTEMBER 1, 2020

This can help healthcare professionals identify a SARS-CoV-2 infection in people suspected to carry the virus with results typically ready in 15 minutes. SARS-CoV-2 Rapid Antigen Test package insert [2] European Centre for Disease Prevention and Control. At launch, there will be 40 million SARS-CoV-2 Rapid Tests available, per month.

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Broad Institute

JUNE 27, 2024

Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. They used an engineered adeno-associated virus (AAV) that crosses the blood-brain barrier after intravenous administration.

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Codon

FEBRUARY 13, 2024

doi: 10.2210/rcsb_pdb/goodsell-gallery-048 The Virus that Cures It’s been over 25 years since the science magazine Discover first ran an extraordinary article about how a long-forgotten medical treatment, used in the former Soviet country of Georgia, could save us from the growing threat of untreatable, drug-resistant infections.

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Codon

APRIL 23, 2023

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. 📎 Papers Strands of DNA can be used to build molecular logic gates. If you want to edit a gene inside of a neuron, what do you do?

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Codon

APRIL 23, 2023

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. 📎 Papers Strands of DNA can be used to build molecular logic gates. If you want to edit a gene inside of a neuron, what do you do?

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Vaccine DNA Virus Engineering 52

Codon

APRIL 2, 2023

But now, by studying DNA extracted from microbes in the blood of almost 10,000 healthy people, this paper shows that there is no such thing. Read Transcription factors bind to DNA and control gene expression. Read Switchable hydrophobic pockets in DNA protocells enhance chemical conversion. Nature Microbiology. Meeussen J.V.W.

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Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? DNA vectors in mouse models? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context.

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PLOS: DNA Science

OCTOBER 12, 2023

The package insert for the drug, Elevidys , warns of adverse effects of acute liver injury and inflammation around the heart and muscles. million DNA bases. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. He was treated on a Monday and died that Friday, as his organs shut down.

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The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) has approved CABENUVA (consisting of Janssen’s rilpivirine and ViiV Healthcare’s cabotegravir), the first and only once-monthly, long-acting regimen for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in adults.

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Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” At least, that’s what every textbook says.

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Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. billion units of DNA code are transcribed into more than a hundred volumes, each a thousand pages long, in type so small as to be barely legible.” At least, that’s what every textbook says.

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Therapies RNA DNA Engineering 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. A phage is a virus that infects bacteria.

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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. A phage is a virus that infects bacteria.

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DNA Therapies Engineering Clinical Trials 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.

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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.

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DNA RNA Engineering Vaccine 52

Drug & Device Law

DECEMBER 19, 2022

one HDPE resin to another HDPE resin) of a packaging component that may affect the impurity profile of the drug product. (vi) Changes in a drug product container closure system that controls the drug product delivered to a patient or changes in the type (e.g., C) Establishment of a new master cell bank or seed.

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SugarCone Biotech

APRIL 8, 2025

Lentivirus particles readily package genetic sequences for delivery into cells, particularly if the genetic construct is small, generally less than 5500 nucleotides (5.5 Current development efforts that focus on DNA delivery by LNP in vivo are advancing rapidly; large payloads can be accommodated which is very useful.

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Codon

FEBRUARY 9, 2025

To achieve this, E11 Bio synthesizes protein barcodes (short peptides with unique amino acid sequences), packages them into AAV vectors (engineered, non-harmful viruses for drug delivery), and delivers these into the brain tissue.

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Codon

FEBRUARY 23, 2025

Researcher Jerome Horwitz at the Michigan Cancer Foundation developed it as a potential cancer treatment by chemically-modifying thymine, a DNA building block. Since zidovudine failed to interfere with DNA-to-DNA replication, Ostertag wondered if it could block RNA-to-DNA replication instead. In South Africa, 12.6

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