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.

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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.

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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.

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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.

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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.

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Genetic Disease RNA Genetics Genome 69

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

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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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.

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Genetics Medicine Development RNA 52

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.

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RNA Genetics Gene Editing In-Vitro 52

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.

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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 (..)

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RNA Genetics Medicine 72

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 68

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.

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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 (..)

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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.

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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.

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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

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.

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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.

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Development Genetic Disease Genetics Gene Therapy 130

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.

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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. .

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RNA Genetic Disease Drugs Protein 52

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

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

Scienmag

APRIL 27, 2022

CHAPEL HILL, NC – Inside embryonic cells, specific proteins control the rate at which genetic information is transcribed from DNA to messenger RNA – a crucial regulatory step before proteins are created. Then, organs develop and hopefully function properly.

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Scientist Gene RNA Protein 83

Scienmag

APRIL 27, 2021

Credit: Nigel Michki Neurons result from a highly complex and unique series of cell divisions. For example, in fruit flies, the process starts with stem cells that divide into mother cells (progenitor cells), that then divide into precursor cells that eventually become neurons.

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RNA Scientist Gene Development 98

Scienmag

NOVEMBER 6, 2020

JUPITER, FL–A new strategy for treating a variety of diseases known as RNA-repeat expansion disorders, which affect millions of people, has shown promise in proof-of-principle tests conducted by scientists at […].

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RNA Scientist Research Genetics 60

Scienmag

NOVEMBER 20, 2020

Scientists at Goethe University within the international consortium COVID19-NMR refine previous 2D models The genetic code of the SARS-CoV2 virus is exactly 29,902 characters long, strung through a long RNA molecule. It contains the information for the production of 27 proteins.

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Genome Genomics RNA Protein 70

XTalks

OCTOBER 26, 2021

Givlaari was approved by the European Medicines Agency (EMA) in March 2020 and by the US Food and Drug Administration ( FDA ) in 2019. AHP is a life-threatening genetic condition caused by an inherited deficiency in the metabolic enzyme hydroxymethylbilane synthase (HMBS), also known as porphobilinogen deaminase (PBGD).

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Gene Silencing Gene Clinical Trials Clinical Trials 98

pharmaphorum

DECEMBER 1, 2020

Pfizer and BioNTech have filed their COVID-19 vaccine with the European medicines regulator, paving the way for a potential approval before the end of the year. The EMA has also confirmed it has received a filing from Moderna, which is producing a rival vaccine based on similar RNA technology.

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Vaccination Vaccine RNA Clinical Trials 88

pharmaphorum

AUGUST 10, 2021

The three-year project – led by respiratory medicine expert Dr Timothy Hinks from the Oxford University Respiratory Medicine Unit – will use whole-genome sequencing of around 500 patients with severe asthma, comparing their gene sequences with control subjects who don’t have asthma.

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Gene Sequencing Gene Bioinformatics Genomics 116

Scienmag

NOVEMBER 18, 2020

— Research on a genetic heart disease has uncovered a new and unexpected mechanism for heart failure. This landmark discovery found a correlation between the clumping of RNA-binding proteins long linked to neurodegenerative disease ? ROCHESTER, Minn.

Genetics 40

Genetics RNA Protein Research 40

Scienmag

JANUARY 18, 2021

CopyKAT enables researchers to gain new insights when analyzing solid tumor samples Credit: MD Anderson Cancer Center HOUSTON — In an effort to address a major challenge when analyzing large single-cell RNA-sequencing datasets, researchers from The University of Texas MD Anderson Cancer Center have developed a new computational technique to accurately (..)

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RNA Research Development Bioinformatics 53

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Editing Gene In-Vivo Gene Therapy 147

Scienmag

JULY 9, 2021

Credit: Elisabeth Paymal To splice or not to splice… In an article published in the journal RNA, Karan Bedi, a bioinformatician in Mats Ljungman’s lab, Department of Radiation Oncology at the University of Michigan Medical School, investigated the efficiency of splicing across different human cell types.

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RNA Bioinformatics Genetics Medicine 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. was swooped up by Real Chemistry in January.

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Vaccination Vaccine Gene Therapy RNA 98