Pharmaceutical Technology

FEBRUARY 9, 2023

ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. The company’s VoyageR AI platform integrates computational methods to drug traditionally undruggable RNA targets.

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Development RNA Gene Therapy Genetics 241

Scienmag

MAY 7, 2021

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

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Genetics Genetic Disease RNA Protein 95

Scienmag

OCTOBER 27, 2020

In the world of rare genetic diseases, exome and genome sequencing are two powerful tools used to make a diagnosis. A recent addition to the toolkit, RNA sequencing, has been demonstrated to help researchers narrow down disease candidate variants identified first on exome and genome sequencing.

Genetic Disease 69

Genetic Disease RNA Genetics Genome 69

Camargo

JULY 27, 2021

The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing). Formulation Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

APRIL 20, 2023

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA.

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Gene Therapy RNA Gene Development 240

Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

Genetics 130

Genetics Gene Expression DNA Antibody 130

Scienmag

APRIL 14, 2021

Credit: Surajit Chatterjee To better understand how RNA in bacteria gives rise to protein–and along the way, target these processes in the design of new antibiotics–researchers are turning their attention to the unique way this process happens in bacteria.

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RNA Bacteria Protein Research 82

World of DTC Marketing

DECEMBER 29, 2020

Both the Pfizer and Moderna vaccines copied RNA sequence from the virus genome and found a way to manufacture it at scale with high-level processes and quality control. These vaccines were developed using different “platform technologies” that involve slotting genetic material from the virus into a tried and tested delivery package.

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Vaccination Vaccine Development Clinical Trials 307

Medical Xpress

NOVEMBER 22, 2022

Scientists at University of Florida (UF) Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.

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RNA Scientist Genetics Development 76

Scienmag

JANUARY 22, 2021

Cryo-electron microscopy study allows researchers to visualize structural changes in an E.

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RNA Production Regulation Bacteria 85

Medical Xpress

NOVEMBER 8, 2022

Researchers at the University Hospital Bonn (UKB) and the Technical University (TU) Dresden have investigated an underlying mechanism and deciphered the driving force of uncontrolled interferon production through studying the rare autoimmune disease Aicardi-Goutières syndrome.

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RNA Genetics Medicine Production 98

Scienmag

JULY 19, 2022

A key genetic mutation that occurs early on in cancer alters RNA “dark matter” and causes the release of previously unknown RNA biomarkers for cancer early detection, a new study by UC Santa Cruz researchers published in the journal Cell Reports shows. Kim A key genetic mutation that occurs early on in […].

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RNA Regulation Gene Genetics 64

Scienmag

SEPTEMBER 29, 2021

JUPITER, FL – The American Chemical Society announced that its prestigious 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry honors a student from the Skaggs Graduate School of Chemical and Biological Sciences at Scripps Research and her faculty advisor, for their pioneering work developing RNA-targeting medications for genetic (..)

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RNA Genetic Disease Scientist Genetics 77

Scienmag

NOVEMBER 13, 2020

In a screening for a functional impact to the neuronal differentiation process, Danish researchers identified a specific circular RNA, circZNF827, which surprisingly “taps the brake” on neurogenesis. Credit: Christian Kroun Damgaard and Anne Kruse Hollensen.

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RNA Regulation Protein Production 93

Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry ssRNA virus peptides is a key innovation area in pharmaceutical Single-stranded RNA (ssRNA) viruses establish a platform for viral replication in the cytoplasm during the early stages of infection and efficiently generate mature virus.

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RNA Genetics Vaccination Vaccine 240

Scienmag

NOVEMBER 11, 2020

Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […]. .

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Protein Genetics RNA Development 98

Medical Xpress

APRIL 13, 2023

A new clinical RNA sequencing platform at The Hospital for Sick Children (SickKids) is helping to facilitate research into rare genetic conditions and carve a path for Precision Child Health, a movement at SickKids to deliver individualized care for every patient.

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RNA Genetics Research 69

Scienmag

JULY 2, 2021

Studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers Credit: UC San Diego Health Sciences In cancer research, it’s a common goal to find something about cancer cells — some sort (..)

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RNA Protein Drugs Research 87

XTalks

FEBRUARY 24, 2025

Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.

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Trials RNA Allergies Drugs 59

pharmaphorum

JANUARY 26, 2023

Both long- and short-read sequencing today is faster, more affordable, and highly accurate, providing researchers with deep insights to fuel research. Genomic insights will increasingly be used to reduce the hundreds of millions of pounds wasted administering drugs that are ineffective due to an individual’s genetic makeup.

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Genome Genomics Genome Project Genetics 129

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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RNA Gene Therapy Genetics Genetic Disease 130

Medical Xpress

DECEMBER 29, 2022

A clinical research team from the University of Hong Kong (HKUMed) has used amniotic fluid cells obtained during 16-24 weeks of pregnancy as a novel sample type for RNA-sequencing in prenatal diagnosis to help more families with tailored clinical management. The findings have been published in the journal, npj Genomic Medicine.

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RNA Genomics Genome Clinical Research 71

Pharmaceutical Technology

MAY 25, 2023

The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

Gene Therapy 243

Gene Therapy Gene Gene Editing Development 243

Scienmag

MARCH 9, 2021

CHOP researchers find small changes in RNA due to RNA editing could have a large impact on gene expression and phenotype Philadelphia, March 9, 2021–Combining computational mining of big data with experimental testing in the lab, researchers at Children’s Hospital of Philadelphia (CHOP) have identified RNA editing events that influence (..)

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Gene Expression RNA Gene Big Data 53

Worldwide Clinical Trials

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

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Genetics Protein Gene Genotype 195

pharmaphorum

MAY 14, 2021

Biogen has made a further push into RNA-based drug discovery via a collaboration with Envisagenics, which applies artificial intelligence RNA sequencing data to discover new drug targets and therapeutics. At least 370 genetic disorders are known to derive from errors introduced during this splicing process.

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RNA Licensing Licensing Genetics 60

Roots Analysis

AUGUST 17, 2023

Genome sequencing has historically been classified into DNA sequencing and RNA sequencing. There are several RNA sequencing service providers coming in this market. In order to overcome these challenges, several stakeholders are focused on employing cutting edge technologies that can examine the quantity and sequences of RNA samples.

RNA 52

RNA DNA Genome Genomics 52

XTalks

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. The frequent intravenous infusions can be challenging for patients.

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FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

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Development Genetic Disease Genetics Gene Therapy 130

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. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Engineer Genetics Clinical Research Research 52

Outsourcing Pharma

FEBRUARY 24, 2022

The pharmaceutical companyâs Lilly Institute for Genetic Medicine reportedly will focus on developing RNA-based therapeutics in a âstate-of-the-artâ facility.

Genetics 52

Genetics Medicine Development RNA 52

Pharmaceutical Technology

MARCH 28, 2023

The system comprises the Cas9 enzyme and a guide RNA. The company will also be eligible to receive research and development milestones of up to an additional $230m, along with royalties on any future products resulting from the deal. It edits genes by precisely cutting DNA, then allowing natural DNA repair processes to take over.

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Gene Editing DNA Gene Gene Therapy 235

Scienmag

SEPTEMBER 17, 2020

Coleen Murphy found the key to multi-generational pathogen avoidance: a small RNA called P11 Credit: Illustration by Rachel Kaletsky, Rebecca Moore and Coleen Murphy When humans see their children about to eat something they oughtn’t, we can simply tell them, “Don’t eat that. It’ll make you sick.”

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RNA Research Gene Genetics 98

Medical Xpress

JANUARY 18, 2023

Researchers at Columbia Engineering and Columbia University Irving Medical Center have invented a new RNA sequencing method that achieves high-quality results from small volumes of frozen tumor specimens. The paper was published January 9, 2023, by Nature Genetics.

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RNA Engineer Genetics Research 87

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

Life Science 162

Life Science Development Gene Therapy Genetics 162

Scienmag

DECEMBER 16, 2020

New research from the Whitehead Institute suggests that the products of transcription — RNA molecules — regulate their own production through a feedback loop Credit: Jon Henninger/Whitehead Institute At any given moment in the human body, in about 30 trillion cells, DNA is being “read” into molecules of messenger RNA, the intermediary (..)

RNA 52

RNA DNA Regulation Production 52

Scienmag

NOVEMBER 6, 2020

A compound designed at Scripps Research works in early tests against myotonic dystrophy 1 and Fuchs endothelial corneal dystrophy, which together affect millions of people.

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RNA Scientist Research Genetics 60