The Open Targets Blog

JANUARY 30, 2025

gget ( [link] ) is a free, open-source command-line tool and Python package designed to enable efficient querying of large genomic databases, such as Ensembl, UniProt, and NCBI. Joe is a fourth-year USC-Caltech MD-PhD student in the Pachter Lab, and he is working on a novel algorithm to detect carcinogenic variants in RNA sequencing data.

Bioinformatics 77

Bioinformatics Immune Response RNA Packaging 77

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. Qualified commercial RNA extraction chemistries include Perkin Elmer, Thermo Fisher, Roche, and Qiagen. ABOUT SPECTRUM SOLUTIONS AND SPECTRUM DNA.

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RNA DNA Engineering Virus 52

The Pharma Data

MARCH 7, 2022

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. Cas9 binds and uses RNA as an address-tag. The AI-designed blocking protein was the cargo of the dCas9-RNA construct.

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RNA DNA Packaging Small Molecule 52

Broad Institute

JUNE 27, 2024

CRISPRoff silences the targeted gene by adding methyl groups, chemical tags that prevent the gene from being transcribed or read into RNA and so from being expressed as protein. The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body.

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Disease Therapies Clinical Trials Engineering 142

The Pharma Data

DECEMBER 8, 2020

Laying down a new track for RNA processing, Remix launched with $81 million in financing. Funds will be used to support development of the REMaster technology platform and advance the company’s pipeline of RNA processing targeted therapeutics as well. “As Pear Therapeutics. Most read today on BioSpace: Source link.

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RNA Therapies Disease Protein Expression 52

Codon

JULY 27, 2023

A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges. How do transcription factors couple up with the correct RNA molecule?

Therapies 52

Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges. How do transcription factors couple up with the correct RNA molecule?

Therapies 52

Therapies RNA DNA Engineering 52

Codon

APRIL 2, 2023

Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). By combining this new variant with standard sequencing methods, it’s possible to identify RNA modifications in a really simple way. Nucleic Acids Research. Nature Biotechnology.

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DNA RNA Engineering Licensing 52

The Pharma Data

DECEMBER 16, 2020

Gustave Perna, who oversees the logistics for Operation Warp Speed, told reporters earlier this week that McKesson will package and distribute the vaccine to more than 3,000 sites across the country. The Moderna vaccine is similar to Pfizer and BioNTech’s vaccine in the sense that they both use an approach utilizing messenger RNA (mRNA).

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Vaccine Immune Response FDA FDA Approval 52

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Read more in Nature Methods.

DNA 52

DNA RNA Engineering Vaccine 52

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Read more in Nature Methods.

DNA 52

DNA RNA Engineering Vaccine 52

Codon

APRIL 2, 2024

The RNA Exporter , which was developed by Felix Horns and others in my lab, is one example. It came from the idea that everyone studies RNA and it’s useful to look at the transcriptome and how it changes over time, but these tools all require that you kill the cell. The exported RNA could transfect just the target cell.

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RNA Engineering Molecular Biology Synthetic Chemistry 56

Codon

MARCH 24, 2024

In the case of semaglutide, those cells are Saccharomyces cerevisiae— also known as Baker’s yeast — engineered to secrete a peptide precursor that is later purified, chemically modified, packaged into an injectable or tablet form, and then shipped around the world. Continuing this method may not scale.

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Engineering Vaccine Production RNA 86

The Pharma Data

JANUARY 17, 2021

It received FDA guidance January 5 th and is in the midst of preparing the first package of investigational new drug (IND) applications – usually with an n-of-1, for individual patients. That’s not the case with RNA-targeted drugs. Patient dosing is expected to begin later this year. “No

Disease 52

Disease Clinical Trials Small Molecule Treatment 52

The Pharma Data

MARCH 20, 2021

Coronaviruses are RNA viruses and include common human coronaviruses, severe acute respiratory syndrome-CoV (SARS-CoV), Middle East respiratory syndrome-CoV (MERS-CoV) and SARS-CoV-2 ( 1 ). INTRODUCTION. SARS-CoV-2 nucleocapsid is highly expressed during infection ( 22 ) and a potential target for viral detection by mass spectrometry.

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Virus Assay Development Laboratories RNA 52

The Pharma Data

SEPTEMBER 1, 2020

In general, antigen tests have a high specificity, though are not as sensitive as molecular tests that amplify the target viral DNA or RNA sequence in order to generate a quantifiable signal to indicate the presence of the virus in a sample. SARS-CoV-2 Rapid Antigen Test package insert [2] European Centre for Disease Prevention and Control.

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Virus Laboratories Clinical Trials Treatment 52

Codon

JUNE 26, 2023

A T4 phage can hold 171,000 bases of DNA or other molecules, including proteins and RNA. For context, the AAV9 that is often used in gene therapies has a packaging limit of 4,700 DNA bases.) It uses single-molecule RNA fluorescence to measure mRNAs and fluorescent reporters to measure the proteins. Paper by Sha G. in Nature.

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DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

A T4 phage can hold 171,000 bases of DNA or other molecules, including proteins and RNA. For context, the AAV9 that is often used in gene therapies has a packaging limit of 4,700 DNA bases.) It uses single-molecule RNA fluorescence to measure mRNAs and fluorescent reporters to measure the proteins. Paper by Sha G. in Nature.

DNA 52

DNA Therapies Engineering Clinical Trials 52

The Pharma Data

AUGUST 3, 2021

The company confirmed the tirzepatide SURPASS program has met global regulatory submission requirements for evaluating cardiovascular risk and its intention to submit the registration package to regulatory authorities by the end of 2021. Business Development/Other Developments.

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Production Marketing Research DNA 52

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. In the ATLAS study, CABENUVA met the primary endpoint for noninferiority (the proportion of participants with plasma HIV-1 RNA ?50 50 c/mL, meeting noninferiority criteria.

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FDA Approval Treatment RNA FDA 52

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? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context.

Therapies 59

Therapies RNA Protein Expression DNA 59

The Pharma Data

MAY 3, 2021

Pfizer reported that it submitted its application for extension of authorization last week, and EMA announced today that it has undertaken the evaluation of the messenger RNA (mRNA) vaccine. The vaccine has been authorized by the EU for use in people 16 years and up since December 2020. .

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Vaccine RNA Clinical Trials Packaging 52

The Pharma Data

NOVEMBER 30, 2020

Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced that the primary efficacy analysis of the Phase 3 study of mRNA-1273 conducted on 196 cases confirms the high efficacy observed at the first interim analysis.

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Vaccine FDA Clinical Trials Disease 52

The Pharma Data

MAY 13, 2021

Stimulates DNA and RNA synthesis. You should apply the MindBody Matrix Pain Relief cream 3-4 times per day for the first week after you receive your package. Increases your energy levels by increasing ATP production. Activates and speeds up the lymphatic system. Reduces painful symptoms associated with arthritis.

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Pharmacy Doctors Production Therapies 52

Codon

OCTOBER 27, 2024

tuberculosis known as rpoB , which codes for part of its RNA polymerase. This test is still only a proof-of-concept, and the engineers will have to contend with the constraint that Raman spectroscopy relies on complex equipment when they come to package the test as diagnostic.

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Immune Response Doctors Disease Laboratories 85

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. Stahl et al. on bioRxiv. If you want to edit a gene inside of a neuron, what do you do? These fat bubbles are injected straight through the skull.

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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. Stahl et al. on bioRxiv. If you want to edit a gene inside of a neuron, what do you do? These fat bubbles are injected straight through the skull.

Vaccine 52

Vaccine DNA Virus Engineering 52

Codon

NOVEMBER 1, 2024

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. SeqFISH, developed by Long Cai’s group at Caltech, maps the spatial positions of thousands of RNA, DNA, or protein molecules within single cells.

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DNA RNA Molecular Biology Laboratories 114

Codon

FEBRUARY 23, 2025

.” In 1974, Wolfram Ostertag , a German geneticist at the Max Planck Institute, explored whether zidovudine could block retroviruses , which carry RNA, and use a reverse transcriptase enzyme to convert it into DNA before integrating into the host genome.

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Clinical Trials Virus Vaccine DNA 65