Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. mRNA contains instructions that direct the cells to make proteins.

Research 59

Research Gene Therapy RNA Gene 59

Delveinsight

SEPTEMBER 23, 2021

Boehringer stated that the Abexxa’s generation aims at cancer-specific proteins situated throughout the mobile versus proteins at the mobile membrane broadening the selection of possible cancer antigen objectives. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Jeffrey Stafford, Ph.D.,

RNA 61

RNA Gene Therapy Gene Protein 61

XTalks

JANUARY 3, 2024

In individuals affected by ATTR, which includes both hereditary and wild-type (non-hereditary) variants, the TTR protein forms fibrils that accumulate in various tissues. These tissues comprise peripheral nerves, the heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Also, exosomes are mostly being used for the detection of circulating RNA and microRNA. “An

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks. This system is designed to deliver RNA editing technology or other payloads directly to particular body areas, such as the nervous system or muscle. and Leila Zegna, director of the Kabuki Syndrome Foundation.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

XTalks

NOVEMBER 30, 2023

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

DECEMBER 1, 2021

The country of South Africa has a particularly sophisticated infectious disease monitoring, surveillance and research infrastructure, which is why the country has been at the forefront in both the identification of coronavirus variants as well as COVID-19 vaccine trials.

Gene 105

Gene Genome Genomics Genetics 105

pharmaphorum

DECEMBER 1, 2020

The companies initially filed the nonclinical data and other technical data, followed by emerging clinical trial results. In the trial, BNT162b2 also showed an overall favourable tolerability with no safety concerns reported to date. The first primary objective analysis was based on 170 confirmed cases of COVID-19.

Vaccination 88

Vaccination Vaccine RNA Clinical Trials 88

pharmaphorum

NOVEMBER 18, 2021

The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity and rare diseases. billion in cash. The transaction is expected to close in the fourth quarter of this year.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

Drug Discovery World

MARCH 21, 2023

In July 2022, DDW reported that molecular genetics company Mainz Biomed had enrolled its first patient in an international clinical study assessing the potential combination of gene expression (mRNA) biomarkers with a diagnostic test for colorectal cancer.

Drugs 52

Drugs Gene Expression RNA Clinical Trials 52

pharmaphorum

OCTOBER 6, 2020

In the case of a rolling review, the EMA evaluates trial data as they become available before a complete filing is submitted. The vaccine is based on different technology from AZ’s rival: while AZ’s uses a weakened and genetically modified cold virus to prime the immune system, BNT162b2 is based on a string of RNA.

Vaccination 59

Vaccination Vaccine RNA Immune Response 59

Delveinsight

JANUARY 7, 2021

The immunotherapy pipeline is led by an in-licensed EP4 antagonist that Ikena is testing combined with Keytruda in phase 1b/2 colorectal and non-small cell lung cancer clinical trials. Eli Lilly backed Terns Pharmaceuticals now has two NASH candidates in clinical trials. IND-enabling studies are underway.

RNA 52

RNA Clinical Trials Clinical Trials Bioavailability 52

pharmaphorum

NOVEMBER 9, 2020

Top-line results come from a phase 3 trial of the candidate known as BNT162b2 and an evaluation of 94 cases from the trial of 43,538 patients. Results will be discussed with regulators worldwide and the European Medicines Agency has already begun its rolling review of data to hasten a decision on the clinical trial dossier.

Vaccination 98

Vaccination Vaccine Clinical Trials Clinical Trials 98

Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment.

RNA 98

RNA Genome Genomics Engineer 98

Drug Discovery World

JANUARY 23, 2023

“Put simply, radioresistant GBM cells prefer to stock up on fatty acids instead of utilising them as an energy source in order to reduce mitochondrial reactive oxygen species that may cause damage to their DNA, RNA, and proteins, and, in turn, cell death,” explains Prof Youn.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

pharmaphorum

NOVEMBER 23, 2021

The bolt-on deal comes – which includes an upfront payment of $120 million and up to $910 million in milestones – covers rights to ARO-HSD, Arrowhead’s RNA interference drug targeting an enzyme called HSD17B13 that seems to contribute to the pathology of NASH and other liver diseases.

Sales 52

Sales Drugs Trials RNA 52

pharmaphorum

DECEMBER 21, 2020

Their technology is based on synthetic messenger RNA – short for ribose nucleic acid – which is a short transcript of a longer DNA code. As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein.

Vaccination 111

Vaccination Vaccine Doctors Doctor 111

The Pharma Data

DECEMBER 8, 2020

Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. The company’s product candidates consist of novel human cells engineered to produce the crucial proteins, enzymes or factors needed by patients living with chronic diseases such as hemophilia, diabetes and lysosomal storage disorders.

RNA 52

RNA Antibody Life Science Protein 52

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. Its job is to carry coding information that is essential to the translation and processing of functional proteins.

RNA 52

RNA Protein Vaccination Vaccine 52

The Pharma Data

JANUARY 10, 2021

The new year began with a fairly low level of clinical trial news. Arcturus Therapeutics got the FDA go-ahead for its Phase II trial of its COVID-19 vaccine candidate ARCT-021. The trial will enroll 600 participants, with 450 receiving ARCT-021 and 150 receiving placebo. Here’s a look. COVID-19-Related. Non-COVID-19-Related.

Gene Therapy 52

Gene Therapy Trials Gene Clinical Trials 52

XTalks

APRIL 6, 2022

XTALKS WEBINAR: Using CRISPR interference (CRISPRi) Viability Screens to Map Long Noncoding RNA Dependencies in Tumor Cells. Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

DNA 105

DNA In-Vitro Doctors Doctor 105

The Pharma Data

OCTOBER 19, 2020

With over 300 active clinical trials, Winship researchers are dedicated to taking new cancer treatments from bench to bedside. cancer centers selected as a Lead Academic Participating Site for the NCI’s National Clinical Trials Network (NCTN). Winship is one of 32 U.S. MBA, FACP, Chairman of the Caris Precision Oncology Alliance.

Life Science 52

Life Science Clinical Trials Clinical Trials RNA 52

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

The Pharma Data

JULY 6, 2021

Hemophilia is a rare, genetic bleeding disorder in which a person’s blood does not clot properly. New research will also be presented on Eloctate ® Antihemophilic Factor (Recombinant), Fc Fusion Protein, Sanofi’s extended half-life factor VIII therapy for people with hemophilia A. ePoster Abstract # OC48.2

Protein 52

Protein RNA Marketing Development 52

STAT News

MARCH 6, 2023

Cambridge-based Moderna recently reported encouraging results from a study of another type of personalized cancer vaccine it developed with the pharmaceutical giant Merck; it uses messenger RNA technology to target advanced melanoma. Neoantigen vaccines are designed to target certain proteins called neoantigens on tumor cells.

Vaccination 98

Vaccination Vaccine Drugs Clinical Trials 98

Delveinsight

AUGUST 6, 2020

Transthyretin (TTR) is a protein that the liver produces to facilitate the transfer of thyroid hormone and vitamin A in the blood. However, due to specific reasons, the TTR protein begins to misfold and aggregate as fibrils, a process known as amyloidosis of Transthyretin (ATTR). hATTR Market: Present Treatment Options.

Gene Silencing 52

Gene Silencing Protein Marketing Gene 52

Bioengineer

JULY 23, 2021

. “For the first time, we identified a crucial interaction between platelets and the tumor via their surface proteins,” Jain said. ” Jain’s team in College Station for this research includes postdoctoral researcher Dr. . ” ###.

Engineer 52

Engineer Gene Editing Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 30, 2021

Overall, Leqvio was well-tolerated, with a safety profile similar to placebo and consistent with the overall pooled population from the combined trials 1 – 3. As the first and only small interfering RNA to provide effective and sustained LDL-C reduction, Leqvio helps manage a critical cardiovascular risk factor for ASCVD.

RNA 52

RNA Trials Production Containment 52

Drug Discovery World

OCTOBER 4, 2022

The pandemic might have affected the number of clinical trials taking place for cell and gene therapies, with activity decreasing by around 15% compared to 2020, but major therapies such as Novartis’ gene therapy drug for spinal muscular atrophy (SMA) Zolgensma still made it to market. .

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

Drug Discovery World

SEPTEMBER 28, 2022

AI-based diagnostic software and clinical trials technology proved to be two of the most attractive health investments, alongside mental health and wellness and medical imaging companies. . According to Hampleton Patners’ latest Healthtech M&A market report2, digital health companies raised a total of $57.2

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines.

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

DECEMBER 8, 2020

chief executive officer at Ionis, said, “In 2020, we pursued an aggressive agenda focused on building our commercial plans and capabilities, progressing the Ionis-owned pipeline, advancing our technology and growing our leadership position in RNA-targeted therapeutics. Monia , Ph.D., It is available here for a limited time.

Medicine 52

Medicine RNA Production Marketing 52

XTalks

AUGUST 30, 2022

Clinical research experts from the CRO Medpace shared insights about the operational and regulatory considerations for neuroscience trials with direct CNS administration. Such therapies may involve small interfering RNA (siRNA) or oligonucleotides in viral transfection platforms, large molecules and antibodies.

Trials 83

Trials Drug Delivery Drugs Gene 83

XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

Life Science 98

Life Science RNA Vaccination Vaccine 98

Roots Analysis

JANUARY 28, 2024

Pharmaceutical Research: Big data analytics can be applied in various areas of pharmaceutical research, including drug discovery and development, precision medicine , clinical trial optimization, pharmacovigilance and drug safety, supply chain optimization, and real-time monitoring and surveillance.

Big Data 40

Big Data Medicine Marketing Clinical Trials 40

The Pharma Data

DECEMBER 18, 2020

The Moderna COVID-19 Vaccine contains messenger RNA (mRNA), which is genetic material. The vaccine contains a small piece of the SARS-CoV-2 virus’s mRNA that instructs cells in the body to make the virus’s distinctive “spike” protein. The vaccine was 94.1%

Vaccination 52

Vaccination Vaccine Production Manufacturing 52

pharmaphorum

JUNE 28, 2021

Preliminary results from a phase 1 trial run by Regeneron and CRISPR specialist Intellia Therapeutics – co-founded by Nobel Prize winner Jennifer Doudna – showed steep reductions in a biomarker of ATTR amyloidosis disease activity with a single dose of the NTLA-2001 drug. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105