Pharmaceutical Technology

JUNE 11, 2024

Discover the groundbreaking patent by Silence Therapeutics Plc for a compound revolutionizing RNA interference therapy. Learn how formula (II) is paving the way for targeted treatment of liver disease and genetic disorders.

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pharmaphorum

SEPTEMBER 6, 2024

Eli Lilly signs another genetic medicines deal, this time with Genetic Leap, a start-up that applies AI to the discovery of RNA-targeted drugs.

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Bio Pharma Dive

NOVEMBER 12, 2024

Trace Neuroscience aims to advance an RNA-binding therapy designed to preserve — and potentially improve — muscle function in people living with the nerve-destroying disorder.

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RNA Genetics Medicine 173

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.

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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. 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

Pharmaceutical Technology

NOVEMBER 8, 2022

Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Together with Neurophth, we will develop the world’s best AAV ophthalmic gene therapy products and bring brightness back to patients all over the world.”.

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Gene Therapy Gene Development Genetics 305

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

BioSpace

OCTOBER 15, 2023

The next frontier in RNA therapies, RNA editing has the potential to treat both genetic and common disorders, and the technology is rapidly expanding beyond the liver.

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Bio Pharma Dive

AUGUST 9, 2023

The Flagship-backed company claims its technology can create genetic drugs that work broadly to treat diseases caused by “stop” mutations.

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RNA Drugs Genetics 130

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

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

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.

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Pharmaceutical Technology

MARCH 22, 2024

is revolutionizing Parkinson's disease treatment with RNA molecules and CRISPR technology. Learn about their patent for precise genetic modifications. Discover how AnGes Inc.

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RNA Genetics Gene 130

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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Medical Xpress

MAY 15, 2023

Irregularities in the body's genetic coding to make proteins are linked to cancerous tumors. But most genetic material contains elements whose function isn't clear.

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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

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.

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Scienmag

JANUARY 22, 2021

coli enzyme synthesizing ribosomal RNA that shift it between turbo- and slow-modes depending on the bacteria’s growth rate Credit: Murakami Laboratory, Penn State The enzyme that makes RNA from a DNA template is altered to slow the production of ribosomal RNA (rRNA), the […].

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Pharmaceutical Technology

JUNE 8, 2023

DTx-1252 is a fatty acid ligand conjugated oligonucleotides (FALCON) small interfering RNA (siRNA) therapeutic which represses the PMP22 gene in Schwann cells. DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. “We

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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

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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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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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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Medical Xpress

NOVEMBER 8, 2022

The innate immune system fights infectious agents, and the alarm messenger interferon plays a central role in this. However, if interferon is produced in the absence of an infection, it can lead to autoimmune diseases.

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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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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

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

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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Scienmag

NOVEMBER 11, 2020

“Conditional gene knockout and small interfering RNA […]. 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.

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Protein Genetics RNA Development 98

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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BioPharma Reporter

OCTOBER 17, 2024

Wave Life Sciences reports that it has successfully carried out RNA editing in two patients with alpha-1 antitrypsin deficiency, the first time this kind of clinical RNA editing has been completed in humans according to the company.

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RNA Genetic Disease Genetics Life Science 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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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

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 Genome Genomics Clinical Research 75

Scienmag

AUGUST 4, 2020

Led by Boyce Thompson Institute’s Andrew Nelson, four partners will identify RNA modifications and develop resources that may lead to hardier crops Credit: Photo credit: Anna Nelson Dittrich ITHACA, NY, August 4, 2020 — RNA perform a variety of functions in cells, helping with everything from regulating genes to building proteins.

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