Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

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Broad Institute

JANUARY 8, 2024

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Researchers have used a variety of methods to deliver these molecular machines to cells, including lipid nanoparticles and viruses.

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Broad Institute

OCTOBER 23, 2024

Machine learning guides researchers to new synthetic genetic switches By Tom Ulrich October 23, 2024 Breadcrumb Home Machine learning guides researchers to new synthetic genetic switches A new method allows precise activation or repression of genes in specific cells and tissues. By Sarah C.P.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.

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Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

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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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Drug Target Review

AUGUST 22, 2023

This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What is the current landscape of treatment options for Huntington’s disease, and how does this potential therapy fit in?

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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. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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Drug Target Review

JANUARY 2, 2024

In research published in Scientific Reports , 1 investigators focused on mesenchymal stem cells (MSCs), known for their potential in treating cell defects and regulating immune responses. Our recent work proposes the use of an intracellular toolkit to map organelle bio-geography in stem cells that could lead to more precise therapies.”

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RNA Immune Response Therapies Disease 128

Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. At the heart of any research is the scientific question. This acts as a safeguard against false positives and misinterpretation of the data.

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RNA Drugs Research Science 52

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).

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RNA Bioinformatics Pharmacokinetics Science 59

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.

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

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Chemical Biology and Drug Design

APRIL 1, 2023

Many kinds of research are being focused on the cytotoxic activity of berberine in in vitro and in vivo studies. Berberine anticancer properties are demonstrated due to the interaction of berberine with micro-RNA. Besides this, it induces cell death by apoptosis, autophagy and necrosis in cancer cells.

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Codon

NOVEMBER 3, 2024

Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB. Chilean research participants aged 15 to 35 who received the vaccine during a case-control study , for example, only showed a 10 percent reduced risk of pulmonary TB.

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Vaccine Trials Therapies Treatment 96

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027.

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DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs.   The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach.

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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.

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Broad Institute

DECEMBER 6, 2023

A new, large-scale reference created by researchers at the Broad Institute of MIT and Harvard could help scientists and clinicians better understand the role of cytokines in health and disease. AC: This research could have far-reaching implications, accelerating our understanding of natural immune responses and human immune-related diseases.

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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. The study appears in Nature Biotechnology.

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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.

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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Chemical Biology and Drug Design

AUGUST 24, 2023

Abstract Targeted therapy has attracted more and more attention in cancer treatment in recent years. However, due to the diversity of tumor types and the mutation of target sites on the tumor surface, some existing targets are no longer suitable for tumor therapy.

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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.

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Engineering Protein Production RNA Vaccine 140

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. We will replace it.”

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Small Molecule RNA Clinical Trials Drugs 52

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

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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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Broad Institute

JUNE 28, 2023

Today, scientists use these reactions to produce customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and many aspects of basic biomedical research. “I Longer synthetic DNA and RNA molecules are also critical for modern biologic drugs.

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Alta Sciences

FEBRUARY 19, 2025

Researchers must characterize the anti-drug-antibody (ADA) response in preclinical and clinical studies and report any ADA-positive samples as a risk-based approach. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

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Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

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Codon

APRIL 9, 2023

This is Codon Digest, my weekly roundup of research, news, and industry highlights about the future of biology. ” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. RNA may offer a safer form of pest control in the future.

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DrugBank

JULY 24, 2024

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases.

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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.

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Drug Target Review

JULY 7, 2023

This gives epigenetic editing the unique ability to treat complex multigenic diseases and opens the door to enhancing cell and regenerative therapies, which require the manipulation of multiple genes for optimal results. Author Bio: Dr Blythe Sather Dr Blythe Sather is the Vice President, Head of Research at Tune Therapeutics.

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Fierce BioTech

MARCH 14, 2025

Join us for an exclusive webinar exploring groundbreaking findings from the DUTRENEO trial, which evaluated the role of predictive biomarkers in guiding neoadjuvant therapy for muscle-invasive bladder cancer (MIBC). Don't miss this opportunity to gain deep insights from leading experts in oncology and biomarker research.

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Chemical Biology and Drug Design

NOVEMBER 27, 2023

To clarify how A6 affected on MCF-7 cells, RNA-seq analysis was conducted to evaluate the whole genome effect of A6 on gene expression. These results indicate that AchEI A6 could be a potential antitumor agent for breast cancer patients and could help the development of novel therapies.

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