Drug Discovery World

JANUARY 17, 2023

The research aims to develop solutions for the limitations associated with adeno-associated virus (AAV), the current primary means of delivery for gene editing. . AAV has limited packaging capacity compared to LNPs and it can prompt an immune system response,” Sahay said. “It

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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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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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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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Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing.

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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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Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g., CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.

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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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addgene Blog

MAY 30, 2023

Adeno-associated virus (AAV) is a single stranded, Parvoviridae DNA virus, packaged in a non-enveloped icosahedral capsid, that can be used to express genes of interest in cell and animal models.

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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

Drug Discovery World

FEBRUARY 8, 2023

This helps ensure a high concentration of loaded capsids and adequate removal of impurities, such as host cell proteins and DNA. The threat of empty capsids Capsids are the protein shell that would usually enclose the genetic material of a virus. Pharmacology of Recombinant Adeno-associated Virus Production. 2021;23:254-262.

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Drug Discovery World

DECEMBER 15, 2022

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Reduce/eliminate chromosomal disruptions caused by double-strand DNA breaks. In one example, Choi et al.

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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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DNA RNA Engineering Licensing 52

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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Broad Institute

DECEMBER 13, 2023

The studies are part of a package of 10 papers in Nature that take distinct, yet complementary, approaches to mapping the mouse nervous system at the single-cell level. The package also includes the spatial, single-cell atlas of the mouse brain and spinal cord that was led by Wang and Liu and was first published online in September in Nature.

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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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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

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

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

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 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

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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