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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RNA Vaccine Small Molecule Therapies 133

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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Science Daily: Pharmacology News

MAY 29, 2024

New research has determined the spatial structure of various processes of a novel gene-editing tool called 'prime editor.' Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix.

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RNA DNA Therapies Treatment 119

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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Research Immune Response Clinical Trials Therapies 137

Science Daily: Pharmacology News

JULY 27, 2023

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

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RNA Research Therapies Disease 72

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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Therapies RNA Regulations Research 100

BioPharma Drive: Drug Pricing

JUNE 3, 2024

The alliance hands Lilly an RNA-based therapy in preclinical testing for ALS and frontotemporal dementia, and includes a research collaboration to develop other, similar medicines.

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RNA Therapies Drugs Research 87

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.

Therapies 52

Therapies Research Clinical Trials 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.

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Engineering Protein Production RNA Vaccine 140

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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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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RNA Medical Schools Engineering DNA 98

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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DNA RNA Disease Hospitals 128

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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RNA Therapies Drugs Treatment 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. 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.

Research 52

Research RNA Molecular Biology Small Molecule 52

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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DNA RNA Science Therapies 98

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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Therapies RNA Pharma Companies Disease 56

Broad Institute

JANUARY 22, 2024

At the Broad Institute of MIT and Harvard and elsewhere, researchers are also learning how dependencies affect cancer cells and how they influence each other and contribute to drug resistance. How have researchers looked for cancer dependencies in the past? There are a number of limitations to those approaches.

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Drugs Therapies Regulations RNA 128

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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Therapies RNA Engineering Vaccine 52

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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Treatment RNA Hospitals Disease 96

Chemical Biology and Drug Design

FEBRUARY 26, 2024

The m 6 A-RNA immunoprecipitation (MeRIP) assay detected the m 6 A modification profile. In all, METTL3 induced miR-4654 maturation in a m 6 A-dependent manner, which was then reduced SOD2 expression, thus promoting apoptosis and oxidative stress in HLECs, suggesting a novel path for DC therapy.

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RNA Regulations Therapies Research 100

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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DNA Engineering Therapies Regulations 105

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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Bioinformatics RNA Regulations Therapies 100

Codon

JULY 27, 2023

For a recent study, researchers edited 21 different genes in poplar trees, a species commonly used to make paper. The researchers studied trees with 70,000 different gene-editing combinations, just 0.5% But what happens if you restore these cone cells, using gene therapy? Max Berry left an excellent comment. Gene-edited trees.

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Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

For a recent study, researchers edited 21 different genes in poplar trees, a species commonly used to make paper. The researchers studied trees with 70,000 different gene-editing combinations, just 0.5% But what happens if you restore these cone cells, using gene therapy? Max Berry left an excellent comment. Gene-edited trees.

Therapies 52

Therapies RNA DNA Engineering 52

Broad Institute

AUGUST 19, 2024

A team of researchers at the Broad Institute of MIT and Harvard; the Ragon Institute of Mass General Brigham, MIT, and Harvard; and St. Jude Children’s Research Hospital have discovered that oleic acid, one of the most abundant fatty acids in the body, restores a healthy balance of vaginal microbes in a laboratory model of BV.

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Hospitals Treatment Medical Schools Science 121

Broad Institute

DECEMBER 13, 2023

By Leah Eisenstadt December 13, 2023 Two teams of Broad researchers produced detailed maps of the mouse nervous system. Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs.

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DNA Virus Research Engineering 140

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Research also took place in two facilities in Italy, with sponsorship from Italfarmaco S.p.A. Researchers have been working on developing gene therapy for DMD for decades. million DNA bases.

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FDA Approval Treatment FDA DNA 98

The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Therapies Laboratories Molecular Biology RNA 52

Drug Target Review

SEPTEMBER 4, 2023

Infectious agents’ resistance was described at the European Union Research Parliament “as a widespread, multifaceted phenomenon that affects both living things and the environment, as well as their capacity to survive in the presence of drugs intended to either kill or inactivate them.”

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Small Molecule Therapies Regulations Treatment 105