RNA editing: emerging from CRISPR’s shadow
BioPharma Drive: Drug Pricing
OCTOBER 22, 2024
Early study data from Wave Life Sciences suggests how editing RNA may yield viable medicines. Large and small drugmakers say such results are just the start.
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Wave sees RNA editing validation in early trial results
BioPharma Drive: Drug Pricing
OCTOBER 16, 2024
The results provide the first clinical evidence of RNA editing, a burgeoning field that's drawn interest from biotechs and pharmaceutical companies alike.
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RNA that doesn't age
Science Daily: Pharmacology News
APRIL 5, 2024
Certain RNA molecules in the nerve cells in the brain last a life time without being renewed. RNAs are generally short-lived molecules that are constantly reconstructed to adjust to environmental conditions. Neuroscientists have now demonstrated that this.
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Orna, a circular RNA specialist, acquires a buzzy startup
BioPharma Drive: Drug Pricing
MAY 23, 2024
After laying off staff late last year, Orna is expanding through the purchase of ReNAgade Therapeutics, which was built around technology designed to more effectively deliver RNA-based medicines.
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Research shows how RNA 'junk' controls our genes
Science Daily: Pharmacology News
JULY 2, 2024
The new study focuses on critical snippets of RNA in the tiny, transparent roundworm Caenorhabditis elegans (C. The study provides a detailed map of the 3'UTR regions of RNA in C. 3'UTRs (untranslated regions) are segments of RNA involved in gene regulation.
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Bayer partners with RNA drugmaker to develop new cancer therapies
BioPharma Drive: Drug Pricing
AUGUST 28, 2024
Under a collaboration with NextRNA Therapeutics, Bayer will access the biotech’s platform to target long, non-coding RNA interactions with small molecule drugs.
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Judo Bio debuts with $100M and a plan to take RNA drugs to the kidney
BioPharma Drive: Drug Pricing
OCTOBER 7, 2024
The Cambridge, Massachusetts-based biotech is using what it calls ligand-siRNA conjugates to reach drug targets in the kidney, which historically have been difficult to reach with RNA therapies.
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Researchers develop RNA-targeting technology for precisely manipulating parts of human genes
Science Daily: Pharmacology News
JUNE 24, 2024
Researchers have harnessed a bacterial immune defense system, known as CRISPR, to efficiently and precisely control the process of RNA splicing.
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RNA: The most attractive target in recent viral diseases
Chemical Biology and Drug Design
DECEMBER 13, 2023
This up-to-date commentary/perspective article sheds light on the revolutionary approaches designed to target the principal viral weapon “RNA” used by RNA viruses (e.g., the severe acute respiratory syndrome coronavirus 2 “SARS-CoV-2”) to infect humans and spread infections, the genomic RNA strands.
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RNA processing in health and disease: challenges and opportunities of the field
Drug Target Review
JUNE 15, 2023
In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.
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Engineers design more powerful RNA vaccines
Science Daily: Pharmacology News
SEPTEMBER 7, 2023
By adding synergistic self-adjuvanting properties to Covid-19 RNA vaccines, researchers showed they could significantly boost the immune response generated in mice.
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RNA for the first time recovered from an extinct species
Science Daily: Pharmacology News
SEPTEMBER 19, 2023
A new study shows the isolation and sequencing of more than a century-old RNA molecules from a Tasmanian tiger specimen preserved at room temperature in a museum collection. This resulted in the reconstruction of skin and skeletal muscle transcriptomes from an extinct species for the first time.
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New technique illuminates how abnormal RNA splicing leads to disease
SCIENMAG: Medicine & Health
AUGUST 14, 2023
A technique that enables scientists to record gene mutations and patterns of gene activity in individual cells has been extended to cover RNA splicing as well, in a study led by researchers at Weill Cornell Medicine, the New York Genome Center and the Princess Margaret Cancer Centre in Toronto.
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Roche partners with RNA editing biotech Ascidian
BioPharma Drive: Drug Pricing
JUNE 18, 2024
While the Swiss pharma recently pruned its neurology drug pipeline, it has shown interest in the possibilities of RNA-focused approaches like what Ascidian is exploring.
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New imaging method enables detailed RNA analysis of the whole brain
Science Daily: Pharmacology News
NOVEMBER 21, 2024
Researchers have developed a groundbreaking microscopy method that enables detailed three-dimensional (3D) RNA analysis at cellular resolution in whole intact mouse brains. The new method, called TRISCO, has the potential to transform our understanding of brain function, both in normal conditions and in disease, according to the new study.
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Agents that target viral RNA could be the basis for next generation broad spectrum anti-viral drugs– new study
Drug Discovery Today
MAY 10, 2021
A new approach to tackling viruses by targeting the ‘control centre’ in viral RNA could lead to broad spectrum anti-viral drugs and provide a first line of defence against future pandemics, according to new research at the University of Birmingham.
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The Access to Advanced Health Institute receives $18 million award to develop a temperature stable, single-dose chikungunya RNA vaccine through a phase 1 clinical trial
SCIENMAG: Medicine & Health
AUGUST 3, 2023
The Access to Advanced Health Institute Receives $18 Million Award to Develop a Temperature Stable, Single-Dose Chikungunya RNA Vaccine Through a Phase 1 Clinical Trial Credit: Delaney Brown Photography The Access to Advanced Health Institute Receives $18 Million Award to Develop a Temperature Stable, Single-Dose Chikungunya RNA Vaccine Through a Phase (..)
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Merkin Prize in Biomedical Technology awarded to F. William Studier for development of widely used protein- and RNA-production platform
Broad Institute
MAY 14, 2024
William Studier for development of widely used protein- and RNA-production platform By Corie Lok May 14, 2024 Breadcrumb Home Merkin Prize in Biomedical Technology awarded to F. Merkin Prize in Biomedical Technology for his development of an efficient, scalable method of producing RNA and proteins in the laboratory.
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Molecular biologists identify framework for understanding RNA editing in a disease-causing parasite
SCIENMAG: Medicine & Health
JULY 19, 2023
Together, they have published dozens of papers on the mechanics of mitochondrial DNA and RNA in a single-celled, disease-causing parasite called Trypanosoma brucei. As molecular biologists at Boston University and as husband and wife, Ruslan Afasizhev and Inna Afasizheva, have worked together for decades.
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Biotech startup Airna raises $60M for RNA editing medicines
BioPharma Drive: Drug Pricing
JULY 31, 2024
Launched last September, the company is working on a treatment for alpha-1 antitrypsin deficiency, which it said could enter the clinic in 2025.
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Roche joins startup Remix in bet on another way to drug RNA
BioPharma Drive: Drug Pricing
JANUARY 3, 2024
The deal is the third Roche has formed with startups developing pills that target RNA, following similar pacts with Arrakis and Ribometrix.
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Wave readies clinical testing for first RNA editing therapy
BioPharma Drive: Drug Pricing
SEPTEMBER 5, 2023
The biotech, one of several advancing RNA editing medicines, expects to obtain “proof-of-mechanism” data in patients with the inherited disorder alpha-1 antitrypsin deficiency by next year.
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Roche bets on Ionis RNA medicines with neuroscience deal
BioPharma Drive: Drug Pricing
SEPTEMBER 27, 2023
The Swiss drugmaker is restocking its pipeline by buying into preclinical Ionis medicines for Alzheimer’s and Huntington’s diseases.
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Ipsen, Skyhawk to develop RNA-targeting pills for rare brain diseases
BioPharma Drive: Drug Pricing
APRIL 22, 2024
billion, is the latest research collaboration involving small molecule drugs that modify RNA. The deal, which could be worth up to $1.8
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Choosing the Right RNA Analysis Methods: Genome-wide RNA-Seq vs. Nanostring nCounter vs. Targeted RNA-Seq Panels
Crown Bioscience
JULY 15, 2024
In the world of RNA analysis, researchers have an array of technologies at their disposal. In this post, we'll delve into three popular RNA analysis methods— transcriptome-wide RNA-Seq, Nanostring nCounter, and targeted RNA-Seq panels—comparing their features and discussing when to use each.
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RNA interference and its role in Alzheimer’s disease
Drug Target Review
JANUARY 24, 2024
Scientists from Northwestern Medicine have demonstrated that RNA interference could have a crucial role in the onset and development of Alzheimer’s disease (AD). sRNAs Cellular functions rely on numerous protein-coding and noncoding RNAs and the RNA-binding proteins associated with them, which form ribonucleoprotein complexes (RNPs).
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Single Cell RNA Profiling FFPE Tissue
Fierce BioTech
NOVEMBER 5, 2024
Single Cell RNA Profiling FFPE Tissue Explore the robust cross-platform agreement between scRNA-seq and bulk RNA-seq, ensuring consistent and reproducible data on immune cell presence. Quality control metrics highlight the optimal conditions for processing FFPE curls, with the highest DV200 samples yielding the most robust data.
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A covalent compound selectively inhibits RNA demethylase ALKBH5 rather than FTO
Covalent Modifiers
FEBRUARY 25, 2024
TD19 irreversibly modifies the residues C100 and C267, preventing ALKBH5 from binding to m6A-containing RNA. Moreover, TD19 displays good anticancer efficacy in acute myeloid leukemia and glioblastoma multiforme cell lines.
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Startup AIRNA emerges from stealth to bring RNA editing to a rare disease
BioPharma Drive: Drug Pricing
SEPTEMBER 19, 2023
AIRNA is one of several biotechs to debut plans for target alpha-1 antitrypsin deficiency in recent years, with competitors such as AlveoGene, Wave Life Sciences and Arrowhead Pharmaceuticals.
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Beyond the lab: RNA
Drug Target Review
DECEMBER 6, 2023
Dear readers, RNA, or ribonucleic acid, plays a pivotal role in the intricate dance of cellular processes. We are committed to providing you with the highest quality content, and we believe that this report will deliver a different perspective on the topics covered.
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An RNAi renaissance is creating a new generation of startups
BioPharma Drive: Drug Pricing
NOVEMBER 6, 2024
Pioneers like Alnylam Pharmaceuticals have led the RNA interference field for years. Now, a crop of young biotechs is building on that foundation by taking the drugmaking technology in new directions.
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Researchers uncover new CRISPR-like system in animals that can edit the human genome
Broad Institute
JUNE 28, 2023
They showed that Fanzor proteins use RNA as a guide to target DNA precisely, and that Fanzors can be reprogrammed to edit the genome of human cells. The new study demonstrates that RNA-guided DNA-cutting mechanisms are present across all kingdoms of life. said Zhang.
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Decoding stem cells for personalised regenerative medicine
Drug Target Review
JANUARY 2, 2024
Forging the toolkit for subcellular omics Cellular components known as organelles, consisting of RNA and protein, exert vital influences on human wellbeing and ailments by sustaining equilibrium, managing growth and ageing, and producing energy.
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RNA editing specialist Korro to go public via reverse merger
BioPharma Drive: Drug Pricing
JULY 14, 2023
The deal with Frequency Therapeutics is the latest biotech reverse merger as private drugmakers still face challenges going public via an IPO.
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The critical role of comprehensive RNA sequencing in liquid biopsy for biomarker discovery and clinical trials
BioPharma Drive: Drug Pricing
JUNE 17, 2024
Elevate precision medicine: dose optimization and immune monitoring through advanced liquid biopsies.
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Novartis buys a preclinical biotech and its RNA drug technology
BioPharma Drive: Drug Pricing
JULY 17, 2023
For $500 million, Novartis will acquire DTx Pharma and its preclinical neurological disease drugs, marking the Swiss company’s latest investment in gene-silencing medicines.
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Life from a drop of rain: New research suggests rainwater helped form the first protocell walls
Science Daily: Pharmacology News
AUGUST 21, 2024
billion years ago, a critical step in the transition from tiny beads of RNA to every bacterium, plant, animal, and human that ever lived. New research shows that rainwater could have helped create a meshy wall around protocells 3.8
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A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis
Broad Institute
APRIL 4, 2024
A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis By Rose Circeo April 4, 2024 Breadcrumb Home A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis The Primer on Medical and Population Genetics is a series of weekly lectures on genetics topics related to human populations and disease.
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Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine
BioPharma Drive: Drug Pricing
JUNE 25, 2024
The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.
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Age prediction from human blood plasma using proteomic and small RNA data: A comparative analysis
SCIENMAG: Medicine & Health
JUNE 30, 2023
“[…] we see our work as an indication that combining different molecular data types could be a general strategy to improve future aging clocks.” Credit: 2023 Salignon et al. “[…] we see our work as an indication that combining different molecular data types could be a general strategy to improve future aging clocks.”
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Streamlining the Path from Discovery to Patient – Simpler RNA Manufacturing with Aldevron’s Codex® HiCap RNA Polymerase
Fierce BioTech
JULY 1, 2024
Streamlining the Path from Discovery to Patient – Simpler RNA Manufacturing with Aldevron’s Codex® HiCap RNA Polymerase swheeler Mon, 07/01/2024 - 17:17 Wed, 08/07/2024 - 10:00 Resource Type Webinar Kyle Studey, M.S. Codex ® is a trademark of Codexis, Inc. Listing Image aldevron 250x190.png
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New approach may yield modified messenger RNAs for treating a wide range of conditions
Broad Institute
SEPTEMBER 23, 2024
Using what they learned from these experiments, they developed a framework — ligation-enabled messenger RNA-oligonucleotide assembly, or LEGO — that enables researchers to chemically modify the structure of mRNA molecules and influence their interactions with the cell's protein-translation machinery, achieving desired therapeutic effects.
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Tensor factorization + single-cell rna-seq
Broad Institute
AUGUST 8, 2024
Tensor factorization + single-cell rna-seq By lcopelan@broadinstitute.org August 8, 2024 Tensor factorization + single-cell rna-seq Neriman Tokcan UMass Boston MIA About Current Schedule Video Playlist Resources All talks Contact us MIA Talks Search - Any - Fall 2024 Spring 2024 Fall 2023 Spring 2023 Fall 2022 Spring 2022 Fall 2021 Spring 2021 Fall (..)
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