Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

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Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). Reece Armstrong explores the potential and future opportunities for mRNA-based therapies.

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Drug Discovery World

SEPTEMBER 6, 2022

In gene therapy, re-engineered adeno-associated virus (AAV) vectors have been produced using various methods such as machine learning or directed evolution. According to the ASGCT, the gene therapy pipeline increased 16% from Q1 2021 with 3,579 gene, cell, and RNA therapies in development from preclinical through pre-registration stages. .

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Codon

MARCH 20, 2023

Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Marine biofilm engineered to produce current in response to small molecules. Ribozyme-mediated RNA synthesis and replication in a model Hadean microenvironment. Szymczak P. Nature Communications.

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Codon

AUGUST 27, 2024

When doctors sequenced the DNA and RNA found in Alice’s blood and synovial fluid—the liquid that surrounds and lubricates joints—they found abnormally low levels of genes encoding iron-storing proteins and high levels of epidermal growth factor receptor RNA. But DNA alone is not always enough to identify a pathogen.

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The Pharma Data

OCTOBER 21, 2020

The COVID-19 pandemic has highlighted the urgent need for a novel, oral antiviral to treat this highly infectious and often deadly virus,” said Jean-Pierre Sommadossi, Ph.D., Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.”.

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The Pharma Data

JANUARY 19, 2021

In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.

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The Pharma Data

DECEMBER 21, 2020

(Nasdaq:BCRX) today announced that data from part 1 of a clinical trial of its broad-spectrum antiviral, galidesivir, showed that galidesivir was safe and generally well tolerated in patients infected with SARS-CoV-2, the virus that causes COVID-19. It is in advanced development for the treatment of Marburg virus disease.

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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. Communications Biology.

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Codon

APRIL 30, 2023

The company uses a modified virus to deliver multiple copies of the correct form of the beta-globin gene into a patient’s blood-producing stem cells. Read Ribosomes are able to build proteins because they contain a strand of RNA that imbues them with their catalytic power. It’s an easier way to get CRISPR into cells!

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Codon

APRIL 30, 2023

The company uses a modified virus to deliver multiple copies of the correct form of the beta-globin gene into a patient’s blood-producing stem cells. Read Ribosomes are able to build proteins because they contain a strand of RNA that imbues them with their catalytic power. It’s an easier way to get CRISPR into cells!

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Codon

JUNE 11, 2024

Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. Small molecules can randomly transit through the pore and create disruptions, and the polymerase ratchet mechanism is imperfect, occasionally causing DNA to temporarily reverse direction through the pore.

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The Pharma Data

SEPTEMBER 9, 2020

Immunization of non-human primates (rhesus macaques) with BNT162b2, a nucleoside-modified messenger RNA (modRNA) candidate that expresses the SARS-CoV-2 spike glycoprotein, resulted in strong anti-viral effects against an infectious SARS-CoV-2 challenge.

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The Pharma Data

SEPTEMBER 8, 2020

Its broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor T cells, bi-specific checkpoint immuno-modulators, targeted cancer antibodies and small molecules.

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Drug Discovery World

NOVEMBER 15, 2022

It’s now possible to programme cell-free systems to make RNA and proteins that interact with each other,” Dr Laohakunakorn continues. One of the challenges we face with SARS-CoV-2 is that the virus continually evolves, making RNA vaccines and antibody therapies less effective. Engineering biosensors for specific functions .

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The Pharma Data

SEPTEMBER 8, 2020

Its broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor T cells, bi-specific checkpoint immuno-modulators, targeted cancer antibodies and small molecules. For more information, visit. www.merck.com. and connect with us on.

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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.

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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.

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DNA RNA Engineering Vaccine 52

Drug Target Review

JULY 11, 2023

AI’s role in sequencing Bulk BCR RNA sequencing looks at specific B cells circulating in a blood sample that are producing these common antibodies. The challenge that we have using bulk BCR RNA,” she continued, “is that an antibody is made up of four components.

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The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

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Codon

MARCH 26, 2023

Link Experimental Tests of the Virtual Circular Genome Model for Nonenzymatic RNA Replication. Link U1 snRNP increases RNA Pol II elongation rate to enable synthesis of long genes. Link A DNA tumor virus globally reprograms host 3D genome architecture to achieve immortal growth. Link Probing metabolism in an E. Mimoso C.A.

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DNA Engineering RNA Science 52

Codon

APRIL 16, 2023

Compact gene editors , called enOsCas12f1 and enRhCas12f1, are smaller than many existing Cas proteins and can thus be more easily delivered in a single virus for gene therapies. Read An RNA-based system to study hepatitis B virus replication and evaluate antivirals. Comparison of transformations for single-cell RNA-seq data.

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