Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.

Treatment 96

Treatment RNA Hospitals Disease 96

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).

RNA 64

RNA Disease DNA Engineering 64

Covalent Modifiers

APRIL 25, 2024

Kinsella Nature , 2024 [link] WRN helicase is a promising target for treatment of cancers with microsatellite instability (MSI) due to its essential role in resolving deleterious non-canonical DNA structures that accumulate in cells with faulty mismatch repair mechanisms 1 , 2 , 3 , 4 , 5.

DNA 173

DNA RNA Treatment Drugs 173

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 59

Clinical Development Clinical Trials Clinical Pharmacology Trials 59

Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. Figure 1: High-level workflow for early drug discovery Once the raw data has been gathered, the next step is to gain a thorough understanding of the data.

RNA 52

RNA Drugs Research Science 52

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. Apart from variations across cell types, organelle diversity also occurs within individual cells.

RNA 128

RNA Immune Response Therapies Disease 128

Chemical Biology and Drug Design

JUNE 11, 2024

Using RNA-seq and through in vitro and in vivo studies, we determined that brusatol suppresses hepatocellular carcinoma progression by inducing ATF3-mediated ferroptosis. Therefore, our research revealed the biological effect of brusatol treatment and provided ATF3 as a novel therapeutic target and prognostic biomarker for HCC therapy.

RNA 100

RNA Bioinformatics Treatment Therapies 100

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. A gene-based treatment would have to alter many cells to exert a noticeable effect. In 2023, two gene-based treatments became available. Muscle makes up about 40 percent of body weight. million DNA bases.

FDA Approval 97

FDA Approval Treatment FDA DNA 97

Broad Institute

OCTOBER 21, 2024

By Leah Eisenstadt October 21, 2024 Credit: Kevin Middleton, Broad Communications In the Bhattacharyya lab, researchers develop methods to quickly identify the most appropriate treatments for bacterial infections. The findings appear in the Journal of Clinical Microbiology.

RNA 115

RNA Hospitals Laboratories Medical Schools 115

Conversations in Drug Development Trends

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. The MAA file in the E.U.

Treatment 81

Treatment Clinical Trials Small Molecule Therapies 81

Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

Conversations in Drug Development Trends

AUGUST 14, 2024

By: Simran Padam, Medical Director, Medical Affairs Personalized treatment approaches have emerged as pivotal in improving outcomes for hematological cancers. Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness.

Treatment 78

Treatment Therapies Disease Protein Expression 78

Broad Institute

JUNE 18, 2024

By Allessandra DiCorato June 18, 2024 Credit: Jon Arizti-Sanz SHINE, a rapid diagnostic test developed by Pardis Sabeti's lab in 2020, uses paper strips and CRISPR enzymes to identify specific sequences of viral RNA in samples.

Virus 144

Virus RNA Laboratories Medical Schools 144

Chemical Biology and Drug Design

OCTOBER 27, 2023

Circular RNAs (circRNAs) are a unique kind of non-coding RNA and play an important regulatory role in OP. Expression of circ_0005753 in human BMSCs with MEL treatment, clinical specimens diagnosed with OP, either with ovariectomy (OVX)-induced mice, was measured by RT-qPCR.

RNA 100

RNA Treatment Regulations Research 100

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

ASPET

OCTOBER 8, 2024

Androgen deprivation is the standard treatment for prostate cancer (PCa) patients. In this study, we used enzalutamide and metformin in combination to explore the possible rescued efficacy of enzalutamide in the treatment of ENZ-r CRPC. However, the disease eventually progresses as castration-resistant PCa (CRPC).

RNA 100

RNA Treatment Therapies Disease 100

Broad Institute

JULY 11, 2024

Targeted drug treatment leads tumor cells to imitate viral infection By Ari Navetta July 11, 2024 Breadcrumb Home Targeted drug treatment leads tumor cells to imitate viral infection Exploiting "viral mimicry," mIDH1 inhibitors trick tumors into thinking they are infected with a virus.

Treatment 72

Treatment Immune Response Drugs Virus 72

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. New treatment approaches that allow for the use of lower and safer doses of GCs would be highly beneficial.

Disease 100

Disease RNA Treatment Regulations 100

Codon

AUGUST 26, 2024

Remdesivir and other broad-spectrum antivirals work by jamming up RNA-dependent RNA polymerase (RdRp), an enzyme that nearly all RNA viruses use to replicate their genomes. Other efforts to build broad-spectrum antivirals have focused on another conserved feature of viruses: double-stranded RNA.

RNA 102

RNA Virus DNA Vaccine 102

ASPET

AUGUST 10, 2023

In this study, using RNA interference library screening, downstream anti-apoptotic molecular signaling components involved in CA-induced STC2-mediated protection against ethanol-induced apoptosis were investigated.

RNA 100

RNA Regulations Treatment Disease 100

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

Drug Target Review

APRIL 26, 2023

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs.

RNA 98

RNA Medical Schools Engineering Disease 98

Chemical Biology and Drug Design

JANUARY 4, 2024

Meanwhile, circular RNA (circRNA) circ_0001615 has been reported to be involved in the malignant development of CRC. The binding between miR-873-5p and circ_0001615, or LASP1, was predicted by Starbase, followed by verification by dual-luciferase reporter and RNA immunoprecipitation (RIP) assays.

Regulations 100

Regulations RNA Treatment Research 100

Drug Hunter

JUNE 26, 2023

Of note, sepiapterin might be a treatment option for patients who don’t typically respond to BioMarin’s sapropterin (Kuvan), as a phenylalanine reduction of 69% was seen in patients with classic phenylketonuria. Unlike sapropterin, sepiapterin is more active in cells and is also brain-penetrant.

Small Molecule 130

Small Molecule RNA Trials Treatment 130

The Pharma Data

JUNE 14, 2023

Scientists discover small RNA that regulates bacterial infection People with weakened immune systems are at constant risk of infection. Their research findings, published in Nature, can inform the development of future treatments for life-threatening acute infections. They named the small RNA SicX (sRNA inducer of chronic infection X).

RNA 40

RNA Regulations Treatment Research 40

DrugBank

MAY 22, 2024

Consider aminergic GPCRs and kinases —while many drugs hit these proteins, they often affect more than one target, a phenomenon known as polypharmacology, leading to complex treatment profiles and side effects. Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

Small Molecule 96

Small Molecule RNA DNA Pharmaceutical Companies 96

ASPET

OCTOBER 8, 2024

Treatment with amitriptyline significantly protected mucosal injury by preserving the population of goblet cells and increasing the expression of tight junction proteins. Additionally, RNA sequencing analysis pointed to the potential involvement of the TLR pathway in the anti-colitic effects induced by amitriptyline.

RNA 100

RNA Treatment Regulations Research 100

Broad Institute

AUGUST 31, 2023

Finally, we have an animal model with human-genetic validity and robust neurobiological overlap with human patients that can help scientists learn how existing treatments work and potentially help identify new ones,” said first author Zohreh Farsi, a staff scientist in the Sheng lab. “We

RNA 143

RNA Medical Schools Research Science 143

Broad Institute

AUGUST 19, 2024

A common fatty acid may help restore healthy vaginal bacteria after infection By Ari Navetta August 19, 2024 Breadcrumb Home A common fatty acid may help restore healthy vaginal bacteria after infection Treatments using oleic acid, a naturally occurring oil used in cells, could improve treatment of recurrent bacterial vaginosis infections.

Hospitals 121

Hospitals Treatment Medical Schools Science 121

Chemical Biology and Drug Design

JANUARY 24, 2024

MiR-556-3p and Acyl-CoA synthetase long-chain family member 4 (ACSL4) interaction was confirmed via dual-luciferase reporter assay and RNA pull-down assay. Our study showed that BCL treatment alleviated OGD/R-induced SK-N-SH cell apoptosis, inflammation and ferroptosis.

Regulations 100

Regulations RNA Treatment Research 100

Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. Over time, the frequency of resistance gene variants in the population will continue increasing because only the bacteria lacking those variants will die with treatment.

Vaccine 102

Vaccine Trials Therapies Treatment 102

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

DNA 128

DNA RNA Disease Hospitals 128

Broad Institute

DECEMBER 6, 2023

Using single-cell RNA sequencing to analyze gene expression in individual cells, the researchers have found how 86 major cytokines affect 17 immune cell types in mice. For many immune-mediated diseases, there's no cure or treatment. Some patients develop resistance to treatment and we cannot predict who they will be.

Immune Response 138

Immune Response RNA Disease Medical Schools 138

Chemical Biology and Drug Design

DECEMBER 28, 2023

RNA sequencing analysis was conducted to study the gene expression profiles of Formononetin-induced H1975_OR cells. However, the potentials and the mechanisms of Formononetin to counteract the Osimertinib resistance in NSCLC are unclear.

RNA 100

RNA Regulations Treatment Research 100

The Pharma Data

DECEMBER 28, 2020

Triple Treatment Combo Beneficial in BRAF-Mutant CRC. Based on RNA profiling, treatment benefit was not dependent on previously established BRAF subgroups or the consensus molecular subtype. Professional. MONDAY, Dec. 23 in the Journal of Clinical Oncology. Scott Kopetz, M.D.,

Treatment 52

Treatment RNA DNA Disease 52

PLOS: DNA Science

MAY 25, 2023

Now Avidity Biosciences has developed a candidate drug that weds a monoclonal antibody (MAb) to a short piece of RNA – a small interfering RNA (siRNA). This novel class of RNA therapeutics is called Antibody Oligonucleotide Conjugates (AOCs™). Synthesizing a bit of RNA, Levin writes, is the simple part.

Treatment 52

Treatment DNA RNA Clinical Trials 52

Broad Institute

MARCH 22, 2024

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

Engineering 140

Engineering Protein Production RNA Vaccine 140