Pharmaceutical Technology

MARCH 1, 2023

Further, the acceptance of new mRNA vaccines has rejuvenated activity within previously established categories of RNA therapeutics including lifesaving antisense technologies. These include antisense oligonucleotides (ASO), RNA interference (RNAi), and RNA aptamers.

RNA 147

RNA Vaccine Vaccination Genetics 147

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.

RNA 147

RNA Vaccine Vaccination In-Vivo 147

Pharmaceutical Technology

FEBRUARY 9, 2023

The collaboration is aimed at discovering and developing new advanced treatments for genitourinary (GU) tumours as well as other related serious diseases using ReviR’s RNA-targeted technologies. ReviR combines computational and high throughput drug discovery technologies to deliver advanced medicines to patients.

Development 241

Development RNA Gene Therapy Genetics 241

Pharmaceutical Technology

MAY 17, 2023

It will use the precision medicine approach, as well as the deep expertise of Chinook Therapeutics in nephrology and knowledge of Ionis Pharmaceuticals in RNA-targeted therapeutics. The collaboration aims to discover, develop and commercialise an ASO therapy.

Development 246

Development Protein RNA Genetics 246

XTalks

DECEMBER 4, 2024

In the Asia-Pacific market, Jupiter has negotiated partnerships with companies such as Sichuan Kelun and Tianjin Pharmaceuticals, focusing on Jotrol’s integration into traditional Chinese medicine frameworks. International patents valid until 2036 further strengthen its global positioning.

Bioavailability 111

Bioavailability Genetic Disease Marketing RNA 111

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

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.

Gene Therapy 240

Gene Therapy RNA Gene Development 240

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.

Genetics 95

Genetics Genetic Disease RNA Protein 95

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

Medical Xpress

NOVEMBER 8, 2022

Their study is published in the Journal of Experimental Medicine (JEM).

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RNA Genetics Medicine Production 98

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

Scienmag

NOVEMBER 26, 2021

Credit: CHUM The amount of a SARS-CoV-2 genetic material—viral RNA—in the blood is a reliable indicator in detecting which patients will die of the disease, a team led by Université de Montréal medical professor Dr.?Daniel Daniel Kaufmann has found. The finding is published today in Science Advances.

RNA 81

RNA Genetics Medicine 81

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

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.

RNA 240

RNA Genetics Vaccine Vaccination 240

pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. The evolving field of genomic medicine. This is just the beginning; the potential is enormous,” he says.

Genomics 105

Genomics Genome Medicine Development 105

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.

RNA 75

RNA Genome Genomics Clinical Research 75

pharmaphorum

NOVEMBER 1, 2022

14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases.

RNA 52

RNA Genetics Gene Editing In-Vitro 52

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

Scienmag

MARCH 9, 2022

— Each simple RNA virus has a genome, its “native RNA.” RIVERSIDE, Calif. This genome dictates how the virus replicates in cells to eventually cause disease. The genome also has the code for making a capsid, the protein shell of a virus that encapsulates the genome and protects it like a nanocontainer.

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Packaging Packaging Genetics Genome 84

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

APRIL 13, 2021

New findings suggest dietary modification and new molecular targets may control common genetic disorder Credit: UT Southwestern Medical Center DALLAS – April 13, 2021 – A chemical modification of RNA that can be influenced by diet appears to play a key role in polycystic kidney disease, an inherited disorder that is the fourth leading cause (..)

RNA 72

RNA Genetics Medicine 72

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. ” The Institute will be headquartered in 334,000 sq.

Genetics 52

Genetics Medicine RNA Scientist 52

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 (..)

RNA 87

RNA Protein Drugs Research 87

Scienmag

JULY 23, 2021

Jude Children’s Research Hospital study highlights the power of comprehensive whole genome, whole exome and RNA sequencing to better understand and treat each patient’s cancer Credit: St. Jude Children’s Research Hospital St.

Medicine 96

Medicine Genome Genomics RNA 96

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Solid Biosciences stated that the partnership boosts its scientific capabilities along with commercial potential in cardiac therapy.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

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.

RNA 60

RNA Licensing Licensing Genetics 60

Worldwide Clinical Trials

AUGUST 14, 2024

They play a crucial role in tailoring hematology oncology therapeutic strategies to individual patients and have transformed personalized medicine in oncology. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195

Genetics Protein Gene Genotype 195

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.

Development 130

Development Genetic Disease Genetics Gene Therapy 130

pharmaphorum

JANUARY 26, 2023

We are already seeing an increase in projects exploring population genomics in Africa, the Middle East, and Asia, with initiatives including the GenomeAsia100K Project and the Genome Aggregation Database focusing on capturing genetic data of non-European individuals. Just one mutation can lead to a complete change in protein formation.

Genomics 129

Genomics Genome Genome Project Genetics 129

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

RNA 52

RNA Genetic Disease Drugs Protein 52

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA. The work, which began in the early 1990s through studies on the C.

Regulation 52

Regulation Genetics Scientist RNA 52

BioPharma Reporter

AUGUST 12, 2021

The new facility will be home to Precision NanoSystemsâs new global headquarters and include multiple GMP suites for manufacturing RNA therapeutics and vaccines.

RNA 52

RNA Genetics Medicine Manufacturing 52

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 Vaccine Vaccination 98

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 (..)

Gene Expression 53

Gene Expression RNA Gene Big Data 53

Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. In addition, the scientists later found that the HAV needs TENT4A/B for its replication.

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Protein RNA Genome Genomics 130