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

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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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

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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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

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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Clinical Trials Vaccine Trials 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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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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DNA Organic Chemistry Science Virus 116

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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Vaccine Trials Virus RNA 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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Vaccine Trials Immune Response Virus 52

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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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 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

The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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Clinical Trials RNA DNA Science 52

KIF1A

FEBRUARY 4, 2024

In addition to sequencing patients, the researchers looked for antibodies to the HTLV-1 virus in the patients’ spinal fluid. We each have two DNA copies of the KIF1A gene; each copy is called an allele and makes its own RNA, which is then translated into protein. What is an allele specific ASO?

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RNA DNA Disease Trials 52

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. billion doses by the end of 2021.

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Vaccine Clinical Trials RNA Trials 52

The Pharma Data

AUGUST 31, 2020

Detects, confirms and differentiates HIV-1 and HIV-2 infections providing clinicians with critical diagnostic data for personalised management of patients with HIV – from appropriate counseling on disease differences to targeted therapy. Human immunodeficiency virus (HIV) is categorized into two types, HIV-1 and HIV-2.

FDA Approval 52

FDA Approval FDA Virus Disease 52

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. Stephen Malina is Head of Engineering at Dyno Therapeutics, a company using machine learning to design better viral vectors for gene therapy. Cite: Stephen Malina. “Driving Toward Nanopores.”

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DNA Engineering RNA Small Molecule 85

New Drug Approvals

SEPTEMBER 7, 2024

Coronaviruses are enveloped, positive-sense, single-stranded RNA viruses. The genomic RNA of CoVs has a 5′-cap structure and 3′-poly-A tail and contains at least 6 open reading frames (ORFs). Although compounds have been reported to inhibit 3CLpro activity, they have not been approved as coronavirus therapies.

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RNA Virus Therapies Pharmaceuticals 62

The Pharma Data

DECEMBER 19, 2020

Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines today announced that the U.S. Healthcare workers have been on the front lines of the fight against the virus and are an inspiration to us all. 20, 2020 02:44 UTC. CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– Moderna, Inc.,

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Vaccine Long-Term Care Facilities FDA Licensing 52

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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

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Therapies RNA Protein Expression DNA 59

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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FDA Approval Treatment RNA FDA 52

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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Therapies RNA Immune Response Treatment 64

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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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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Therapies Disease Medical Schools Virus 52

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.

Virus 52

Virus Engineering RNA Small Molecule 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.

Therapies 52

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

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.

RNA 52

RNA Pharmaceuticals Virus 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.

DNA 52

DNA RNA Engineering Licensing 52

The Pharma Data

JUNE 30, 2021

In May 2019, the FDA granted Breakthrough Therapy Designation for the development of lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced patients with multi-drug resistance in combination with other antiretroviral drugs.

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