ASPET

SEPTEMBER 6, 2023

Toll-like receptor (TLR) 7 and TLR8 are single-stranded RNA-sensing endosomal pattern recognition receptors that evolved to defend against viral infections. However, aberrant TLR7/8 activation by endogenous ligands has been implicated in the pathogenesis of autoimmune diseases including systemic lupus erythematosus.

Disease 100

Disease RNA Treatment Regulations 100

Drug Target Review

APRIL 7, 2025

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

Clinical Development 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

Broad Institute

JULY 19, 2024

A new study reveals that C1q can also enter neurons, where it influences protein production, and can accumulate within neurons over time. These changes, which suggest that C1q may be important for keeping the brain's proteins in check, were age-specific, with noticeable differences in protein production in adult mice compared to younger ones.

Protein Production 111

Protein Production Hospitals RNA Production 111

SCIENMAG: Medicine & Health

JULY 13, 2023

Transfer RNAs (tRNAs) are among the most common types of RNA in a cell and are indispensable for protein production in all known organisms.

Protein Production 76

Protein Production RNA Production 76

Broad Institute

JUNE 29, 2023

They found key differences between where mRNAs are made and where the corresponding proteins are being translated in several cell types, suggesting that cells are regulating translation in ways that existing transcriptomic methods, which analyze mRNA production, aren’t able to detect. By Sarah C.P. and Virginia W.

Protein Production 105

Protein Production RNA Regulations Science 105

Drug Target Review

DECEMBER 16, 2024

When their drug product is finally ready, they still must undergo multiple days of lymphodepleting chemotherapy along with the added risks of toxicities from pre-treatment. Umojas VivoVec gene therapy platform uses a lentiviral particle system to convert any CAR or binder into an in vivo-generated CAR T-cell product.

Therapies 52

Therapies Clinical Trials Treatment Trials 52

Drug Target Review

DECEMBER 7, 2023

Researchers from the Laboratory of Bacteriology at The Rockefeller University have now found that bacteria sense phages by a defensive response named CBASS (cyclic oligonucleotide-based antiphage signalling system) which detects viral RNA. The novel, hairpin-shaped molecule was named cabRNA for CBASS-activating bacteriophage RNA.

RNA 59

RNA Immune Response DNA Molecular Biology 59

KIF1A

SEPTEMBER 16, 2023

Note: For simplicity I will use the term KIF1A when referring to UNC-104 Post-translational modifications: When we talk about modifications to proteins, it’s usually in the context of mutations – a modification to the KIF1A gene makes altered RNA, which translates into altered KIF1A protein.

Regulations 105

Regulations RNA Disease Research 105

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs. This approach can be personalized for each patient by sequencing the tumor's DNA and RNA to identify unique mutations. One of the most promising mRNA applications in this field is protein replacement therapy.

Vaccine 97

Vaccine Treatment Clinical Trials Therapies 97

The Pharma Data

OCTOBER 27, 2020

The test is planned for commercial launch as a CE-IVD ( in vitro diagnostic) certified product in the European Union in Q1 2021. “Applying 3a’s proprietary enhanced RNA technology to PCR testing was a logical next step in our product development pipeline,” said Dr. Heinrich Jehle, managing director of 3a-Diagnostics.

RNA 52

RNA Production International Compliance 52

Sygnature Discovery

SEPTEMBER 11, 2024

We’ve successfully tackled targets for: Unprecedented membrane proteins Intrinsically disordered proteins Refolding from inclusion bodies Large protein complexes Labelled proteins for NMR studies RNA structures by crystallography Across our capabilities Protein Expression & Purification Vast diversity of proteins produced.

Science 52

Science Compound Screening Protein Expression RNA 52

Broad Institute

OCTOBER 16, 2024

William Studier receives the 2024 Merkin Prize in ceremony at the Broad Institute for developing technology used to produce millions of doses of COVID-19 vaccines The groundbreaking, scalable technology is widely used in laboratories around the world today to efficiently produce large amounts of protein and RNA. Merkin (left) and F.

Vaccine 64

Vaccine RNA Laboratories Molecular Biology 64

The Pharma Data

JANUARY 19, 2021

A range of companies are exploring potential gene editing therapies based on a CRISPR-Cas9 system, and the ability to tune the CRISPR cut could prevent off-target editing or control the degree of a target protein’s production. The authors found that tracr-L redirects Cas9 in S. The authors suggest natural tracr-L regulates autoimmunity.

Research 52

Research RNA Molecular Biology Small Molecule 52

Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

Therapies 137

Therapies FDA Approval Engineering Disease 137

Drug Target Review

JULY 7, 2023

For example, chemical modifications to the guide RNA — responsible for directing the epigenetic machinery to the region of interest — can fine-tune the intensity and stability of transcriptional changes. She is currently focused on using genetic tuning to enhance and develop cell and gene therapy products for clinical impact.

DNA 105

DNA Engineering Regulations Therapies 105

Sygnature Discovery

SEPTEMBER 12, 2024

Structures solved of many different protein classes, including membrane proteins, protein complexes and recently RNA. With capabilities across X-ray Crystallography Crystallisation/co-crystallisation screening of proteins, DNA, and RNA using in-house and commercial screens at various temperature.

Fragment Screening 52

Fragment Screening RNA Protein Production DNA 52

DrugBank

JANUARY 29, 2025

In sickle cell disease, for instance, a missense mutation in the HBB gene , encoding the beta-globin subunit of hemoglobin, leads to the production of abnormal hemoglobin S. Gene therapy strategies targeting oncogene inactivation, such as RNA interference and gene-editing technologies, have shown promise in preclinical and clinical studies.

Therapies 52

Therapies DNA RNA Disease 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Pharmaceuticals 52

Pharmaceuticals RNA Small Molecule Protein Production 52

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Laying down a new track for RNA processing, Remix launched with $81 million in financing. Sigilon Therapeutics . Pear Therapeutics.

RNA 52

RNA Therapies Disease Protein Expression 52

DrugBank

JULY 24, 2024

By inhibiting "passenger" gene products like PRMT5 in MTAP-null cells, researchers can selectively target cancer cells while minimizing harm to healthy cells. Indirectly Targeting "Undruggable" Proteins Small molecules can also target "undruggable" proteins indirectly by modulating the production of these proteins.

Small Molecule 98

Small Molecule Drugs Clinical Trials Therapies 98

Broad Institute

JANUARY 22, 2024

Initially, there were two approaches: small hairpin RNA (shRNA)-based and now CRISPR-based experiments. In a paper we published this September , we focused on the idea that cancers might be susceptible not just to loss of function of certain genes or gene products, i.e. having too little gas, but also gain of function, or too much gas.

Drugs 128

Drugs Therapies Regulations RNA 128

PerkinElmer

JULY 13, 2020

Imaging Modalities Help Visualize and Confirm Findings An emerging method of studying viruses is by using high-content imaging solutions to visualize the activity of small interfering RNA (siRNA), also known as short interfering RNA or “silencing” RNA.

RNA 52

RNA Virus Laboratories Science 52

The Pharma Data

OCTOBER 19, 2020

Spectrum’s self-contained saliva collection kit provides critical sample consistency while suspending and neutralizing viral RNA transcripts completely inactivating the live virus. Qualified commercial RNA extraction chemistries include Perkin Elmer, Thermo Fisher, Roche, and Qiagen. ABOUT SPECTRUM SOLUTIONS AND SPECTRUM DNA.

RNA 52

RNA DNA Engineering Virus 52

Broad Institute

JULY 11, 2024

IDH1 normally facilitates the activity of enzymes called demethylases, which remove chemical flags called methylation marks from DNA, allowing genes to be transcribed into RNA. In cells with mutant IDH1, this gene is silenced due to the buildup of 2HG, but mIDH1 inhibitors allow demethylases to facilitate cGAS expression.

Treatment 72

Treatment Immune Response Virus Drugs 72

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. I wanted to use molecular biology to create drugs.

Small Molecule 52

Small Molecule RNA Clinical Trials DNA 52

Sygnature Discovery

SEPTEMBER 12, 2024

We’ve successfully tackled targets for: Unprecedented membrane proteins Intrinsically disordered proteins Refolding from inclusion bodies Large protein complexes Labelled proteins for NMR studies RNA structures by crystallography Across our capabilities Construct Design Structure-guided sequence design. Low endotoxin batches.

Science 52

Science Protein Production RNA Packaging 52

The Pharma Data

DECEMBER 21, 2020

Galidesivir treatment was associated with a more rapid decline in viral RNA levels in the respiratory tract in an apparent dose-dependent manner. About Galidesivir (BCX4430) Galidesivir, a broad-spectrum antiviral drug, is an adenosine nucleoside analog that acts to block viral RNA polymerase.

Clinical Trials 52

Clinical Trials Virus RNA Trials 52

New Drug Approvals

SEPTEMBER 6, 2024

TLR7-9 are among the set that are endosomally located and respond to single-stranded RNA (TLR7and TLR8) or unmethylated single-stranded DNA containing cytosine-phosphate-guanine (CpG) motifs (TLR9). In the case of autoimmune disorders, the aberrant sustained activation of TLR7/8/9 leads to worsening of disease states.

RNA 62

RNA Clinical Trials DNA Disease 62

Broad Institute

SEPTEMBER 11, 2024

High-throughput RNA profiling via up-front sample parallelization. In this role, Narayan leads the somatic cancer genomics product development lab at the Broad Clinical Labs (BCL), a CLIA-certified, CAP-accredited facility providing cutting-edge genomic sequencing services. Clin Cancer Res. 2018;24(8):1872-1880. doi:10.1158/1078-0432.CCR-17-1341

DNA 52

DNA RNA Research Production 52

Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays.

DNA 116

DNA Organic Chemistry Science Virus 116

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.

Vaccine 52

Vaccine Clinical Trials RNA Trials 52

The Pharma Data

JANUARY 31, 2021

Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. About Dynavax.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

The Pharma Data

JANUARY 11, 2021

Moderna is following up on the success of its messenger RNA-based COVID-19 vaccine with plans to develop inoculations for numerous other infectious diseases, the company said Monday. based company has been in business for 10 years, but the COVID-19 vaccine was its first approved product. 18, 2020 ). The Cambridge, Mass.-based

Vaccine 52

Vaccine Clinical Trials Virus RNA 52

DrugBank

MAY 15, 2024

Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. These methods could bypass the need to directly target these proteins with traditional drugs.

Small Molecule 98

Small Molecule Molecular Biology Drugs Disease 98

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

This may also help to derisk parallel product development amongst multiple products for the same condition, where another product in development may gain traditional approval or another product already on the market under accelerated approval may confirm clinical benefit prior to completion of clinical trials to support accelerated approval (e.g.,

FDA 64

FDA Clinical Trials Trials Therapies 64

The Pharma Data

DECEMBER 8, 2020

Potential to launch 6+ new products in next five years. Ionis also has plans to expand opportunistically to new products in additional treatment areas such as hematology, endocrinology and pulmonology. Prioritizing and preparing its growing Ionis-owned pipeline for commercialization. CARLSBAD, Calif. ,

RNA 52

RNA Pharmaceuticals Disease Treatment 52

Drug Target Review

AUGUST 16, 2024

Here, I oversee the medical strategy for both existing products and those in our development pipeline, including outcomes research. Additionally, she develops and fosters relationships with external organisations and experts to ensure that products continue to meet market needs and yield desired patient outcomes.

Medical Schools 111

Medical Schools Nurses Research Production 111