Drug Target Review

AUGUST 9, 2023

14 TDP-43 is a DNA and RNA binding protein that regulates the expression and splicing of several target transcripts. Many potential toxic effects have been suggested, including the mis-splicing of the Stathmin-2 RNA transcript. Projected Increase in Amyotrophic Lateral Sclerosis from 2015 to 2040.” Nature Communications, vol.

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Dark Matter Blog

JANUARY 16, 2023

We are moving rapidly toward escape velocity in our RNA expedition at Arrakis. For those of you joining us for the first time, it’s worth a quick look back at why we decided to go all-in on the RNA world, why people thought we were crazy, and why we know we’re not. Our terra firma is small-molecule medicines.

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Drug Target Review

MAY 24, 2024

2 AlphaFold 3 has been built to model DNA, RNA and smaller molecules (ligands). Google DeepMind’s new AI can model DNA, RNA, and ‘all life’s molecules’. There are several technologies and specific tools being rolled out in this area. The latest exciting advancement is AlphaFold 3, which was just released. Alphafold 3.0:

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Dark Matter Blog

JULY 19, 2021

Back in 2015, I felt compelled to set out in a new direction in drug discovery. However, in June I happened to attend the Gordon Research Conference on Chemical Biology and High-throughput Chemistry where I saw a session on small molecules and RNA. In 2015, Jim was CSO at Rana Therapeutics, but I had heard that he might be moving on.

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Dark Matter Blog

JUNE 21, 2023

Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.

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Small Molecule RNA Pharma Companies Science 52

Broad Institute

MARCH 5, 2024

In this talk, we are motivated by unsupervised problems that arise in the analysis of single cell RNA sequencing data, where sample splitting does not allow us to avoid double dipping. Unfortunately, in some problems, sample splitting is unattractive or impossible.

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Drug Target Review

APRIL 9, 2024

In a previous study, we showed that these peptides decorate all cellular RNAs and thus impair the binding of RNA-binding proteins to RNA. Since 2015 Oscar is also the Vice Director of CNIO as well as Director of its Molecular Oncology Programme, and professor at the Karolinska Institute in Sweden.

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RNA Cell Biology Clinical Development Disease 64

LifeSciVC

DECEMBER 5, 2024

combinatorial chemistry, human genetics, functional genomics, mRNA, I/O, CRISPR, RNA editing, and many others). Over the past decade (2015-2024), looking only at meaningful exits above $100M values: M&As of private biotechs have had a median and average of $270M and $600M, respectively.

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Marketing RNA Science Drugs 76

Broad Institute

OCTOBER 24, 2023

DCD-FG uses a Gaussian non-linear low-rank structural equation model and shows significant improvements compared to state-of-the-art methods in both simulations as well as a recent large-scale single-cell RNA sequencing data set with hundreds of genetic interventions.

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NIH Director's Blog: Drug Development

SEPTEMBER 29, 2015

Next, to see how their tiny models of the human brain respond to various chemicals, the researchers exposed the neural tissue chips to 60 compounds—about half with known neurotoxic effects, and the rest considered harmless to the brain—and measured the activity of 19,000 genes in each organoid by RNA sequencing. 2015 Sep 21.

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Dark Matter Blog

JULY 19, 2021

Arrakis has gone through many significant transitions since its founding in 2015. Or should I say back to retirement, as he was comfortably relaxing at home back in 2016 when Jen Petter lured him back into the fray with the siren call of developing a new class of RNA-targeted small-molecule medicines here at Arrakis.

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The Pharma Data

NOVEMBER 22, 2020

The two companies have been in a strategic research collaboration agreement since 2015, focused on using CRISPR Cas-9 to discover new potential treatments. “Then the RNA plus the protein [Cas9] will cut — like a pair of scissors — the DNA at that site, and ideally nowhere else.”.

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Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Read more in Nature Methods.

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DNA RNA Engineering Vaccine 52

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Read more in Nature Methods.

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DNA RNA Engineering Vaccine 52

Codon

AUGUST 27, 2024

When doctors sequenced the DNA and RNA found in Alice’s blood and synovial fluid—the liquid that surrounds and lubricates joints—they found abnormally low levels of genes encoding iron-storing proteins and high levels of epidermal growth factor receptor RNA.

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The Pharma Data

DECEMBER 13, 2020

The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. More information can be found at www.edigene.com.

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Codon

JUNE 11, 2024

Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. In 2015, Oxford Nanopore spun off another company, Metrichor , to provide base-calling algorithms through a web interface.

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DNA Engineering RNA Small Molecule 83

PLOS: DNA Science

MARCH 30, 2023

Comparing genome sequences is important because they provide a 4-letter language in the bases A, C, G, and T (for DNA) or A, C, G, and U (for RNA). The agency started tracking it in 2013, and the spread accelerated in 2015. For those who claim nothing’s being done to prevent future pandemics, please look at GISAID.

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DNA Hospitals Laboratories Virus 52

Codon

MAY 15, 2023

Ratcliff, figured this out back in 2015. Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. They don’t break off. Cell Systems.

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DNA Bioinformatics Engineering Virus 52

Codon

MAY 15, 2023

Ratcliff, figured this out back in 2015. Made from a Cas9 nickase (the histidine at amino acid position 840 is swapped for an alanine) fused to a reverse transcriptase enzyme (which makes DNA from RNA; transcription in reverse), prime editing clinical trials are expected to begin by 2025. They don’t break off. Cell Systems.

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DNA Bioinformatics Engineering Virus 52

Codon

MARCH 12, 2023

The central dogma is often depicted as DNA→RNA→protein, but it’s much more: A biophysical marvel inside the smallest of vessels. Eterna is an online game (there’s also an app) where anyone on the internet can solve puzzles by manipulating RNA sequences and simulating the results. Biology is a Burrito.

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DNA Engineering Therapies RNA 52

NIH Director's Blog: Drug Development

JANUARY 5, 2016

But before diving into our first “new science” post of 2016, let’s take a quick look back at 2015 and some of its remarkable accomplishments. Four of 2015’s Top 10 featured developments directly benefited from NIH support—including Science’s “Breakthrough of the Year,” the CRISPR/Cas9 gene-editing technique. Protein Cell.

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Codon

SEPTEMBER 22, 2024

The first reliable protocols to differentiate human iPSCs into PGC-like cells in vitro were published in 2015. Epigenetic CRISPR tools are targeted to a particular DNA sequence using a guide RNA molecule. A meiotic cell nucleus beneath the microscope. Image provided by the author.

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Codon

APRIL 19, 2023

kilograms of CO 2 and 330 liters of water to make one kilogram of bananas, according to a 2015 study from Ecuador. In 2016, her team demonstrated that, by adding short snippets of DNA or RNA — which can target and “shut down” the MaMADS regulatory proteins — to bananas, it is possible to slow their ripening.

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DNA RNA Engineering Regulations 52

Codon

APRIL 19, 2023

kilograms of CO 2 and 330 liters of water to make one kilogram of bananas, according to a 2015 study from Ecuador. In 2016, her team demonstrated that, by adding short snippets of DNA or RNA — which can target and “shut down” the MaMADS regulatory proteins — to bananas, it is possible to slow their ripening.

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DNA RNA Engineering Regulations 52

Broad Institute

OCTOBER 27, 2024

Our proposed method will be evaluated on synthetic zero-inflated count datasets, simulated single-cell RNA sequencing (scRNA-seq) data, and real-world multi-sample multi-condition scRNA-seq datasets.

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RNA Research 40

Broad Institute

JANUARY 7, 2025

From her first biology course in high school, Rehm loved the logic of genetics: the clear trajectory from a simple code of DNA to RNA to proteins and how disrupting that process could lead to disease. Her mother liked to get things done and made lists for everything, a trait both Rehm and her sister inherited.

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Codon

JANUARY 26, 2025

The 2014-2015 seasonal vaccine, for instance, was only 19 percent effective due to a mismatch between the selected strain and the circulating one. The World Health Organization selects these strains months in advance based on global surveillance data. And their efficacy varies greatly from one year to the next.

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Codon

AUGUST 10, 2023

Nature Reviews Molecular Cell Biology (2015). FEMS Yeast Research (2015). Link A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , by Jinek M. Nature Reviews Genetics (2015). Nature (2015). Nature Protocols (2015). PNAS (2015). Nature Methods (2009). PNAS (1993).

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DNA Cell Biology Molecular Biology Engineering 97

Codon

AUGUST 10, 2023

Nature Reviews Molecular Cell Biology (2015). FEMS Yeast Research (2015). Link A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , by Jinek M. Nature Reviews Genetics (2015). Nature (2015). Nature Protocols (2015). PNAS (2015). & Charpentier E. & Arnold F.H.

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