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The biotech, best known for its vaccine research, said its clinical trial is the first to report results of a messenger RNA therapeutic designed for intracellular protein replacement.
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.
Orbital Therapeutics has raised $270m in a Series A round led by ARCH Venture Partners to advance a portfolio of programmable RNA therapeutics. Orbital will use the new funding to increase the application of RNA-based medicines for use in the fields of new vaccines, immunomodulation and protein replacement.
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.
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 (..)
UK biotech MiNA Therapeutics has signed up another big pharma partner for its small activating RNA (saRNA) platform, which upregulates the activity of proteins, with Eli Lilly the latest to get in on the action. . The post Lilly buys into MiNA’s protein-boosting RNA tech in $1.25bn deal appeared first on.
Flagship Pioneering, the VC fund run by Moderna’s co-founder Noubar Afeyan has launched a new biotech Laronde , with an ambitious plan to create a new class of drugs based on Endless RNA. Called eRNA for short, this class of medicines is programmable and can continuously express therapeutic proteins inside the body.
Perhaps popularized by the COVID-19 vaccines, RNA-based technologies now have the potential to become the next best pesticide to combat crop pests, like insects and fungal pathogens. Over the last year, researchers have been studying the effectiveness of RNA-based pesticides, and there are already a handful of sprays in the works.
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.
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.
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.
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.
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.
The world of drug discovery and chemical probes is still protein-centric and developing highly selective small molecules targeting RNA is often considered to be an insurmountable challenge. Why You Should Attend the RNA- Targeted Drug Discovery Summit. The post 3rd RNA-Targeted Drug Discovery Summit appeared first on.
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. It’s not the first partnership for Biogen in the RNA splicing area.
Now, the HIV research community is setting its sights on vaccines that are in earlier stages of development, based on new approaches like messenger RNA (mRNA) -based vaccines or those using human cytomegalovirus (HCMV) vectors. mRNA vaccines can be made and modified quickly compared to protein subunit vaccines, says Montefiore.
They play a crucial role in tailoring hematology oncology therapeutic strategies to individual patients and have transformed personalized medicine in oncology. Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways.
Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.
Just a few months after breaking cover with its new take on RNA therapeutics, Laronde has raised an impressive $440 million in second-round financing backed by Flagship Pioneering – which was behind the founding of mRNA specialist Moderna. The company has an ambitious goal of producing 100 eRNA medicines in the next decade.
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.
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. .
A subset of fibroblasts located in small foci of tissue on the edges of extensive scarring produce a protein that protects against cell aging. ERJ 2021 Single-cell RNA sequencing has revealed a subset of cells that could provide protection from a rare, but severely debilitating and […].
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. Those specific “regulatory” proteins are called transcription factors, and they do their thing by binding to […].
— 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.
No treatment is currently available to fight this disease, which is due to the overexpression of a specific protein. It affects the peripheral nerves and causes progressive paralysis of the legs and hands. Scientists from the CNRS, INSERM, the AP-HP and the […].
A 2022 study from the Royal College of Physicians and British Pharmacological Society demonstrated the potential of pharmacogenomics, with scientists identifying the genetic cause behind an individual’s drug response for over forty medicines. Just one mutation can lead to a complete change in protein formation.
By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports.
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?
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.
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 biotech is focused on mRNA but has developed a self-amplifying mRNA (sa-RNA) platform in an attempt to develop the potential of the technology further.
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.
Dormitzer comes from Pfizer, where he was chief scientific officer for RNA and viral vaccines for more than six years. AZ said the deal would go beyond Covid-19 and extend into other RNA-based therapeutics. Like Sanofi, Biogen has invested in AI for RNA-based therapeutics this year, announcing a collaboration with Envisagenics.
BioMarin senior vice-president and chief scientific officer Kevin Eggan stated: “Activating RNA therapeutics have the potential to change the way we treat certain genetic diseases, particularly those characterised by the limited production of key proteins.
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.
The AZ vaccine also has a considerable convenience advantage over RNA-based vaccines from Pfizer and Moderna. The body produces antibodies against the protein, which neutralise the coronavirus in the event of an infection. AZ announced the news as reports suggested the UK could approve Pfizer’s rival by the end of the week.
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.
VRG50635 was discovered using Verge’s AI-powered discovery platform ConVERGE which maps out the biological underpinnings of diseases using data on DNA, RNA and protein profiles to identify new targets and drug that can interact with them.
This is the 1st IV infusion of #CRISPR genome editing in patients directly into the bloodstream (target is the liver where the misfolded proteins are made); previously for sickle-cell and beta-thalassemia cells were removed from the body and edited, given back. — Eric Topol (@EricTopol) June 26, 2021.
Not so, says the NIH, which argues that three of its scientists – John Mascola, Barney Graham and Kizzmekia Corbett – should be named as co-inventors of the messenger RNA sequence coding for the SARS-CoV-2 spike protein that is the main component of Moderna’s mRNA-1273 shot. The patent has been filed but not yet awarded.
In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.
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