Drug Target Review

MARCH 21, 2024

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Because mRNA is not a virus, it doesn’t have to meet biosafety requirements for manufacturing and administration that an AAV approach would need.

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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Broad Institute

DECEMBER 6, 2023

Using single-cell RNA sequencing to analyze gene expression in individual cells, the researchers have found how 86 major cytokines affect 17 immune cell types in mice. I hope our approach helps scientists make sense of any immune process, vaccine, disease, or therapy response, so that one can infer which cytokines are contributing to it.

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Broad Institute

MARCH 22, 2024

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

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Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Ribozyme-mediated RNA synthesis and replication in a model Hadean microenvironment. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding.

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The Pharma Data

MARCH 23, 2022

To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.

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Virus Small Molecule Protein Production Research 52

Broad Institute

DECEMBER 13, 2023

The team analyzed the full transcriptome of cells from nearly 100 regions across the mouse brain using high-throughput single-nucleus RNA sequencing, the preferred approach for efforts to create a human brain atlas. In their study, the researchers focused on a variant of an engineered recombinant adeno-associated virus (rAAV) known as PHP.eB.

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DNA Virus Research Engineering 140

Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. Molecular Therapy. Support for $5 per month.

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Therapies RNA Engineering Vaccine 52

Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? 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.

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Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? 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.

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Therapies RNA DNA Engineering 52

The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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Codon

SEPTEMBER 29, 2024

But, regardless of which was first, they all operated with the same core data as their mechanism for understanding life: messenger RNA ( mRNA ). It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Small Molecule Therapies Regulations Treatment 105

Drug Target Review

SEPTEMBER 4, 2023

Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. We discovered it is highly effective and environmentally friendly.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

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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., “Roche shares our passion for delivering innovative new medicines to address great unmet medical needs. Chief Executive Officer and Founder of Atea Pharmaceuticals.

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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Research RNA Molecular Biology Small Molecule 52

Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Therapies Laboratories Molecular Biology RNA 52

Broad Institute

JANUARY 30, 2024

He has also emerged in recent years as a skilled and relatable science communicator, renowned for his efforts at Broad and beyond during the COVID-19 pandemic to unpack the fast-moving research on the new virus in an accessible way and give practical advice about masking, vaccines, and other public health measures.

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The Pharma Data

JANUARY 31, 2021

Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. About Dynavax.

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The Pharma Data

MARCH 8, 2022

The study was published online in advance in the journal Molecular Therapy – Nucleic Acids and is now available in the final version. The virus carries the information to produce a series of proteins, capable of inhibiting antiviral recognition systems of the infected cell. Camouflage protects virus from immune system.

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Clinical Pharmacology Immune Response RNA Virus 52

The Pharma Data

AUGUST 16, 2021

During challenge with the original SARS-CoV-2 virus, animals vaccinated with CV2CoV were found to be better protected based on highly effective clearance of the virus in the lungs and nasal passages. Clearance of the virus in the lungs and nasal passages of the animals was tested following challenge infection with the original virus.

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Immune Response Vaccine Virus RNA 52

The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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Clinical Development Therapies Clinical Trials Treatment 52

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs.   The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach.

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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. 50 c/mL, meeting noninferiority criteria.

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The Pharma Data

DECEMBER 3, 2020

Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. Additionally, Clover is leveraging its in-house GMP biomanufacturing capabilities to support large-scale production of its biologic therapies.

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Drug Target Review

SEPTEMBER 28, 2023

For security reasons, at the time COVID-19 had to be grown in BSL-3 labs, which can be dangerous because they were handling the live virus. These are synthetic, not the live virus, so do not pose a risk of infection. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays.

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The Pharma Data

JANUARY 31, 2021

” Dr. Richard Hatchett , Chief Executive Officer of CEPI, commented: “To end the acute phase of this pandemic, and to control the virus in the longer term, the world needs multiple safe and effective vaccines which can be deployed in a range of populations and countries. About CpG 1018.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.

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Engineering Clinical Research Research Virus 52

The Pharma Data

SEPTEMBER 30, 2021

That’s how easy it’s for SARS-CoV-2, the virus that causes COVID-19, to enter your nose. “We used NanoSTING because the adjuvant for intranasal vaccination and single-cell RNA-sequencing to verify the nasal-associated lymphatic tissue as an inductive site upon vaccination. Breathe in, exhale. But now, we’re one step closer.

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The Pharma Data

JUNE 25, 2021

The outcome confirms that only one single case was attributable to the original SARS-CoV-2 virus. “In addition, the variant-rich environment underlines the importance of developing next-generation vaccines as new virus variants continue to emerge.” More than half of the cases (57%) were caused by Variants of Concern.

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The Pharma Data

JANUARY 21, 2021

The once-a-month regimen was preferred by nine out of 10 patients over the daily oral therapy. While much more remains to be done to make HIV history, today’s milestone reminds us how far medical innovation has come since the first reported cases of the virus almost 40 years ago.”. clinical practices. Most Read Today. Source link.

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FDA Approval Treatment FDA Clinical Trials 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. Gene Therapy. Nucleic Acids Research.

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DNA RNA Engineering Licensing 52

The Pharma Data

NOVEMBER 5, 2020

5 of its ongoing Phase 2 extension study, Study 211, examining vebicorvir (VBR, or ABI-H0731) in patients with hepatitis B virus infection. The company’s other two Phase 2 trials, Study 201 and 202, showed that the addition of VBR to NrtI therapy achieved a quicker, deeper level of viral suppression than with NrtI alone.

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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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The Pharma Data

DECEMBER 10, 2020

In the study, immunization with the hAd5-COVID-19 vaccine inhibited SARS-CoV-2 virus replication in 100% (10 of 10) of Rhesus macaques, with a drop in viral replication starting on the first day of vaccine administration, and undetectable viral levels as early as three to five days post-challenge in most of the animals.

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Vaccine Immune Response Virus Clinical Trials 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. Molecular Therapy.

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DNA RNA Engineering Vaccine 52