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Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system.
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. Antisense therapies are designed for destroying mRNA, reducing the amount of disease-causing protein.
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.
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 […].
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.
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.
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.
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 (..)
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.
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.
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.
Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.
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 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.
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.
— Each simple RNA virus has a genome, its “native RNA.” 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. RIVERSIDE, Calif. This genome dictates how the virus replicates in cells to eventually cause disease.
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.
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.
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.
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.
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.
The company is genetically programming RNA not just to deliver a gene of interest, but to control the location, timing and intensity of therapeutic protein expression using mRNA-encoded logic circuits.
Findings explain how plants use RNA to defend against fungal invaders Credit: Nicole Ward Gauthier/University of Kentucky New research reveals an essential step in scientists’ quest to create targeted, more eco-friendly fungicides that protect food crops.
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.
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.
When cells copy DNA to produce RNA transcripts, they include only some chunks of genetic material known as exons and throw out the rest. The resulting product is a fully-mature RNA molecule, which can be used as a template to build a protein.
The answer may lie in the genetic code of the virus, which scientists at Duke University have found contains several silent mutations that affect protein folding. The researchers focused on the spike proteins that protrude from the surface of the coronavirus, which are responsible for viral attachment and entry into host cells.
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.
Lipids and RNA are two of the essential building blocks of cells. Ribonucleic acid (RNA) is a versatile molecule. On the one hand, like DNA, RNA encodes genetic information which is essential for life to replicate itself. On the other hand, like proteins, RNA molecules carry out chemical reactions.
Transcription is a vital process in bacterial cell, where genetic information in DNA is transcribed to RNA for the translation of proteins that perform cellular function. Hence, transcription serves […].
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. .
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.
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.
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.
Study describes new mechanism for terminating transcription of DNA into RNA in bacteria Credit: Babitzke Laboratory and Dani Zemba, Penn State The protein, known as NusG, pauses the transcription machinery at specific DNA sequences to facilitate what is called “intrinsic termination” and prevent unwanted transcription that could disrupt (..)
RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Super-resolution RNA imaging in live cells.Ribonucleic acid (RNA) is key to various fundamental biological processes. It transfers genetic information, translates it into proteins or supports gene regulation.
In a statement , the Nobel Assembly said the laureates discovered a new class of non-coding RNA molecules that play a crucial role in gene regulation. To activate these instructions, cells create a copy called messenger RNA (mRNA), which exits the nucleus and directs the cell’s machinery to produce a specific protein.
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?
According to the US biopharma company, genetic analyses of publicly available sequences for the new variants of SARS-CoV-2 suggest the mutations in the viral spike protein that have made them more transmissible shouldn’t affect the way Veklury (remdesivir) works. The UK strain – known as B.1.1.7
— 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.
Over the years, pharmaceutical companies have shifted their priorities from traditional interventions towards more advanced pharmacological strategies, such as protein therapeutics. Further, $400 billion is the anticipated sales of protein-based therapeutics in 2023. The mRNA is then translated into to form functional proteins.
Boehringer stated that the Abexxa’s generation aims at cancer-specific proteins situated throughout the mobile versus proteins at the mobile membrane broadening the selection of possible cancer antigen objectives. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Jeffrey Stafford, Ph.D.,
Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases.
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