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.

RNA 52

RNA DNA Vaccine Drug Development 52

ASPET

JULY 19, 2023

Ras- G TPase-activating protein (SH 3 domain)- b inding p roteins (G3BP) are RNA binding proteins that plays a critical role in stress granule (SG) formation. Recent evidence suggests that G3BPs can also regulate mRNA expression through interactions with RNA outside of SGs.

RNA 100

RNA Virus Regulations Disease 100

Drug Target Review

JULY 5, 2023

In nature, proteins take millions and trillions of years to evolve, 1 but scientists do not have this time when designing drugs for life-threatening diseases. Therefore, researchers use directed protein evolution technologies , to evolve and design proteins with specific functions under well-defined conditions and a practical time frame.

RNA 97

RNA Engineering Disease Research 97

Drug Target Review

JULY 24, 2023

Through phylogenetic analysis and microscopy techniques, they identified a nuclear-encoded apicoplast RNA polymerase σ subunit called ApSigma. The study suggests that this regulatory system could be a potential target for future malaria treatments, offering hope for combatting this deadly disease that affects millions worldwide.

RNA 98

RNA Regulations Bioinformatics Laboratories 98

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.

Small Molecule 52

Small Molecule RNA Clinical Trials Drugs 52

Drug Target Review

JULY 11, 2023

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.

Disease 75

Disease RNA Small Molecule Clinical Development 75

The Pharma Data

FEBRUARY 8, 2021

The amended protocol being implemented for all ongoing adult and adolescent fitusiran clinical studies was presented today at the 14 th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

RNA 52

RNA Treatment Vaccine Therapies 52

Chemical Biology and Drug Design

SEPTEMBER 15, 2023

Abstract Chronic rhinosinusitis (CRS) is an inflammatory disease of paranasal sinuses. CRS rabbit models were constructed by Staphylococcus aureus infection and rabbits were injected with lentiviral vectors of short hairpin RNA-targeting Chemerin (shChemerin), followed by micro-computed tomography (CT) scan.

RNA 100

RNA Treatment Disease Research 100

Codon

AUGUST 26, 2024

Credit: OpenStax College The innate immune system has been evolutionarily successful for three reasons: its tools target the most fundamental properties of viruses , act as prophylactics —meaning they prevent diseases, rather than treat them—and are widespread. Existing medicines only pose a partial solution.

RNA 86

RNA Virus DNA Vaccine 86

Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

RNA 98

RNA Regulations Disease DNA 98

KIF1A

SEPTEMBER 16, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This is also important when newborns present as having a “common” disorder that may have a rare genetic component, like KIF1A in genetic cerebral palsy.

Regulations 105

Regulations RNA Disease Research 105

The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.

Treatment 52

Treatment Therapies DNA Trials 52

Broad Institute

AUGUST 19, 2024

Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Jude’s, the team identified a group of genes involved in processing uLCFAs that are present only in the non- Lactobacillus iners species. At the Ragon Institute, Kwon’s lab is working to move this research toward a human clinical trial. “We

Hospitals 121

Hospitals Treatment Medical Schools Science 121

Broad Institute

OCTOBER 28, 2024

So we took lots of reference single-cell RNA sequencing data from atlases and used our scalable machine learning algorithms to embed all of the gene expression data on these cells into compact vector representations — you can think of these as a signature for each cell. We wanted to build a tool like that for cell biology.

Engineering 119

Engineering Cell Biology Disease RNA 119

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

Therapies 98

Therapies Disease Engineering RNA 98

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

Small Molecule 105

Small Molecule Cell Based Assays Disease Assay Development 105

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. The gene presents a complex landscape.

FDA Approval 98

FDA Approval Treatment FDA DNA 98

Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections.

Hospitals 137

Hospitals Medical Schools Virus Science 137

KIF1A

SEPTEMBER 9, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. To investigate these proteins, the authors used RNA interference to silence Nup153 and KIF1A.

RNA 105

RNA Treatment Research Disease 105

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. Please summarise Eterna Therapeutics’ ($ERNA) new research at ASGCT on a cell therapy approach that integrates multiple cell types to emulate the natural immune system’s fight against diseases?

Therapies 103

Therapies DNA Engineering Virus 103

Drug Target Review

APRIL 9, 2024

How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration. However, the mechanism of this observation was not fully understood.

RNA 64

RNA Cell Biology Clinical Development DNA 64

The Pharma Data

MARCH 8, 2021

The data were presented at the 28th Conference on Retroviruses and Opportunistic Infections ( virtual CROI 2021). In patients with chronic kidney disease, assess serum phosphorus. In patients with chronic kidney disease, assess serum phosphorus. Pregnancy and lactation. Source link:[link].

Treatment 52

Treatment Therapies RNA Laboratories 52

Codon

DECEMBER 12, 2024

It’s important to note that the 300-page report, while presenting detailed evidence for the potential harms of mirrored life, uses painstakingly speculative language. DNA and RNA molecules are also built from exclusively right-handed nucleic acids. It gives humanity time to steer away. This is one of them.

DNA 81

DNA RNA Vaccine Virus 81

Drug Target Review

NOVEMBER 13, 2023

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. However, they also limit beneficial responses by suppressing sterilising immunity and limiting antitumour immunity.

Government 104

Government DNA RNA Regulations 104

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. The German study is also scheduled for presentation on Saturday at the American Society of Hematology’s virtual annual meeting.

Therapies 52

Therapies Disease Medical Schools Virus 52

The Pharma Data

NOVEMBER 10, 2021

By creating genetically finagled mouse models and doing single cell RNA-sequencing, Feng and associates plant that Shp2 was needed by excrescence cells for HCC development, which validated and corroborated allowing that Shp2 presented an ideal target for new cancer curatives. ” Source link: [link].

Disease 52

Disease Molecular Biology RNA Drugs 52

DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases. This can potentially correct the root causes of some diseases at the genetic level.

Small Molecule 83

Small Molecule Drugs Clinical Trials Therapies 83

DrugBank

MAY 22, 2024

In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

Small Molecule 67

Small Molecule RNA DNA Pharmaceutical Companies 67

Drug Target Review

SEPTEMBER 25, 2024

5 Here, the original oncogene is still present but the transdifferentiated cancer cells are less dependent on it. Investigating non-oncogene resistance as a parallel defense mechanism used by cancer cells to evade therapies could therefore present a significant opportunity to address drug resistance more comprehensively.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

PLOS: DNA Science

MAY 25, 2023

Myotonic dystrophy type 1 (DM1), an inherited disease affecting muscles, was one of the first described “expanding repeat” disorders. Although it improves symptoms by increasing the ability to relax muscles, it does not affect the underlying cause of the disease. Synthesizing a bit of RNA, Levin writes, is the simple part.

Treatment 52

Treatment DNA RNA Clinical Trials 52

The Pharma Data

DECEMBER 3, 2020

Additionally, “There is not any available single test for the entire stage of the disease,” Gunduz said in a statement. Consequently, performing an immunoassay in the early stages of an infection will be negative because the antibodies cannot be produced yet, even though the disease is present.

RNA 52

RNA Virus DNA Disease 52

Broad Institute

SEPTEMBER 26, 2024

Morphological profiling of cells is commonly employed in drug discovery across many disease areas. However, there has been limited study on its application specifically in the development of therapeutics for heart diseases. The Broad Institute almost doesn’t feel real to me, it is special and unlike any place I've ever visited.

RNA 52

RNA Science Disease Research 52

Codon

AUGUST 27, 2024

Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. Sometimes, though, novel diseases do emerge, and as COVID-19 demonstrated, they can surprise us. This is the third essay of four in our pandemic mini-issue.

DNA 83

DNA Laboratories Hospitals Virus 83

New Drug Approvals

SEPTEMBER 6, 2024

US10071079, Example 15 Ref WO2018005586 [link] The invention further pertains to pharmaceutical compositions containing at least one compound according to the invention that are useful for the treatment of conditions related to TLR modulation, such as inflammatory and autoimmune diseases, and methods of inhibiting the activity of TLRs in a mammal.

RNA 62

RNA Clinical Trials DNA Disease 62

The Pharma Data

OCTOBER 14, 2020

Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Pharmaceuticals 52

Pharmaceuticals RNA Small Molecule Protein Production 52

Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context.

Therapies 59

Therapies RNA Protein Expression DNA 59

Broad Institute

OCTOBER 11, 2023

He also read everything he could about her disease, including emerging evidence that suggested that the immune system could recognize and kill Merkel cell carcinoma. His mother had a presentation of the disease that suggested her immune system was already on the job.

Research 137

Research Immune Response Clinical Trials Therapies 137