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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Genetics RNA Medicine Drugs 104

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 167

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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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

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

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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RNA Genetics 88

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 Genomics 69

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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RNA Genetics Containment Protein 117

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

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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RNA Production Regulation Bacteria 85

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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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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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

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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RNA Genetics Medicine Production 98

XTalks

DECEMBER 4, 2024

CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder. Jupiter joins a wave of biotech IPOs this year, stepping into a competitive space while broadening treatment options for CNS patients.

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Bioavailability Genetic Disease Marketing RNA 111

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

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 Genomics 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

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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RNA Genetics Medicine 72

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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RNA Genetics Gene Editing In-Vitro 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

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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RNA Bioinformatics Protein Gene 74

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 75

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.

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RNA DNA Genome Genomics 52

BioPharma Reporter

AUGUST 1, 2024

AIRNA plans to bring RNA editing therapies to the clinic starting with a treatment for the genetic disease alpha-1 antitrypsin deficiency.

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RNA Genetic Disease Genetics 52

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). We believe the advanced RNA technology and human disease models of Phlox Therapeutics perfectly partner with the delivery tools Solid Biosciences brings to this collaboration.”.

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RNA Gene Therapy Genetics Genetic Disease 130

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

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