LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

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Drug Target Review

JULY 23, 2024

This laid the groundwork for my career in drug discovery using novel modalities like small molecule inhibitors, antibodies, RNA interference (RNAi) and, currently, small molecule protein degraders – which are opportunities to change how we think about medicines.

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Small Molecule Targeted Protein Degradation Science Disease 116

Broad Institute

JUNE 30, 2023

The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds. Typically, studying drug therapies is an expensive and time-consuming undertaking. That teaches us far more than looking for just a single informative data point.”

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. Arrakis is the capstone of my career.

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Dark Matter Blog

JULY 19, 2021

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. Making small-molecule drugs against RNA structures looked like a really cool problem – I want to do THAT! This is a thing?

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Dark Matter Blog

JULY 19, 2021

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. I won’t dwell on Jim’s many accomplishments here.

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Small Molecule RNA Science Therapies 52

Broad Institute

JUNE 21, 2023

Cell Painting uses six fluorescent dyes to image eight different components of a cell: the nucleus, nucleoli, mitochondria, endoplasmic reticulum (ER), golgi apparatus, cytoplasmic RNA, f-actin cytoskeleton, and plasma membrane. Typically, studying drug therapies is an expensive and time-consuming undertaking.

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The Pharma Data

SEPTEMBER 9, 2020

Immunization of non-human primates (rhesus macaques) with BNT162b2, a nucleoside-modified messenger RNA (modRNA) candidate that expresses the SARS-CoV-2 spike glycoprotein, resulted in strong anti-viral effects against an infectious SARS-CoV-2 challenge. billion doses by the end of 2021.

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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. Small molecules can randomly transit through the pore and create disruptions, and the polymerase ratchet mechanism is imperfect, occasionally causing DNA to temporarily reverse direction through the pore.

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DNA Engineering RNA Small Molecule 99

Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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The Pharma Data

MARCH 23, 2022

In fact, antiviral therapies designed to treat viruses such as hepatitis C (Sofosbuvir/SOVALDI) work by blocking viral polymerase. Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase.

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The Pharma Data

JANUARY 17, 2021

As Crooke explained, “With small molecules, if you make any chemical change, you have a new ballgame. That’s not the case with RNA-targeted drugs. Traditional companies have neither the technologies nor the financial incentives needed to develop ultra-rare therapies. We’re the middleman,” he said.

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Disease Clinical Trials Small Molecule Treatment 52

Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. In fact, it was these early pharmaceutical successes that gave us the confidence that we would ultimately succeed in systematically drugging a wide range of RNA structures.

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Small Molecule RNA Clinical Trials Drugs 52

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., SCHEME PATENT PTC Therapeutics Inc.,

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Small Molecule RNA DNA Disease 57

Dark Matter Blog

APRIL 8, 2020

Most people, and in particular most investors we spoke to in those early days, thought we were nuts – that RNA lacked the structural and molecular complexity that medicinal chemistry exploits with such great effect for proteins. One is that molecular recognition is purely a matter of physics and RNA has to play by the same rules.

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RNA Small Molecule Synthetic Chemistry Science 52

Codon

APRIL 2, 2023

Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). By combining this new variant with standard sequencing methods, it’s possible to identify RNA modifications in a really simple way. Gene Therapy. Nucleic Acids Research.

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DNA RNA Engineering Licensing 52

The Pharma Data

OCTOBER 21, 2020

AT-527 is expected to be ideally suited to combat COVID-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses. Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.”.

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Drug Target Review

JULY 11, 2023

AI’s role in sequencing Bulk BCR RNA sequencing looks at specific B cells circulating in a blood sample that are producing these common antibodies. The challenge that we have using bulk BCR RNA,” she continued, “is that an antibody is made up of four components. Cavlan will explore more of these ends in our next article.

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The Pharma Data

MARCH 7, 2022

In their work, Levy and her colleagues focused on a complex of proteins called PRC2 that silences genes by attaching a small molecule, called a methyl group, to a protein that packages genes called histones. Cas9 binds and uses RNA as an address-tag. The AI-designed blocking protein was the cargo of the dCas9-RNA construct.

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RNA DNA Packaging Small Molecule 52

Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Marine biofilm engineered to produce current in response to small molecules. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding.

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Virus Engineering RNA Small Molecule 52

Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

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The Pharma Data

MARCH 4, 2021

We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, Neuroscience, and Gastroenterology (GI). selective GABA A receptor agonist.

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Codon

APRIL 30, 2023

5/ Lots O’ Gene Therapies Not one, but two applications for sickle-cell gene therapies have been submitted to the F.D.A. If approved, these wouldn’t be the first gene therapies on the market, but they would be the first gene therapies for a disease that affects something like 100,000 Americans. Cell Systems.

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Vaccine DNA Engineering RNA 52

Codon

APRIL 30, 2023

5/ Lots O’ Gene Therapies Not one, but two applications for sickle-cell gene therapies have been submitted to the F.D.A. If approved, these wouldn’t be the first gene therapies on the market, but they would be the first gene therapies for a disease that affects something like 100,000 Americans. Cell Systems.

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Vaccine DNA Engineering RNA 52

KIF1A

AUGUST 20, 2024

Susannah is being treated with an Antisense Oligonucleotide (ASO) : To break down the term, this is a molecule designed with a sequence of 20 (oligo, “few”) letters of DNA (nucleotide) that match a sequence on the mutant KIF1A RNA (Antisense) and knock it down. Will this _ KAND?

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The Pharma Data

AUGUST 3, 2021

The company announced donanemab received Breakthrough Therapy designation for treatment of Alzheimer’s disease and its intention to submit a biologics license application (BLA) for donanemab under the accelerated approval pathway later this year based on data from TRAILBLAZER-ALZ. Business Development/Other Developments. billion to $1.1

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Conversations in Drug Development Trends

MAY 30, 2024

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications.

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The Pharma Data

JUNE 26, 2021

” BNT111 is an intravenous therapeutic cancer vaccine candidate encoding for a fixed set of four cancer-specific antigens optimized for immunogenicity and delivered as RNA-lipoplex formulation. Biopharmaceutical New Technologies is a next generation immunotherapy company pioneering novel therapies for cancer and other serious diseases.

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The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

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Codon

APRIL 16, 2023

Compact gene editors , called enOsCas12f1 and enRhCas12f1, are smaller than many existing Cas proteins and can thus be more easily delivered in a single virus for gene therapies. Read Development of a translatable gene augmentation therapy for CNGB1-Retinitis Pigmentosa. Molecular Therapy. Molecular Therapy. Occelli L.M.

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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. Molecular Therapy.

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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. Molecular Therapy.

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DNA RNA Engineering Vaccine 52

The Pharma Data

SEPTEMBER 8, 2020

(LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Cambridge, UK.

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The Pharma Data

SEPTEMBER 8, 2020

About AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.

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Science Vaccine Clinical Trials Pharmaceutical Companies 52

Drug Target Review

FEBRUARY 4, 2025

Currently, there is no cure, and available treatments can only temporarily alleviate symptoms, making the need for new, effective therapies more urgent than ever. These innovative therapies represent a new frontier in Alzheimers care, addressing the critical need for effective treatment options.

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FDA Drugs Disease Science 59

Codon

APRIL 28, 2025

The prevailing hypothesis at the time was that Aequorea jellyfish produced light through luciferin, a small molecule sensitive to oxygen and moisture. Each cell contains messenger RNA (mRNA) that carries instructions for making proteins, so Prasher first extracted mRNA from Aequorea and converted it into complementary DNA (cDNA).

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