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.

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RNA DNA Vaccine Science 52

Drug Discovery World

AUGUST 30, 2024

The investment from Innovate UK will drive both early and later stage innovations in cancer therapies, building on UK economic potential in this field. It is hoped that by interfering with these DNA-binding proteins, these cancers affecting children can be treated.

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DNA Drugs RNA Therapies 147

Broad Institute

JUNE 28, 2023

Merkin Prize in Biomedical Technology for developing an efficient, automated technology for synthesizing DNA. The chemical reactions that he discovered in the early 1980s to accurately and quickly assemble nucleotides into strands of DNA provided an essential element in the development of modern molecular medicine.

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DNA RNA Science Therapies 98

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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Therapies DNA RNA Laboratories 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. DNA vectors in mouse models?

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Therapies RNA Protein Expression DNA 59

Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. The post Why genomic healthcare data matters in the development of new therapies appeared first on Drug Discovery World (DDW). The origins .

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Therapies DNA Disease Research 246

Drug Discovery World

SEPTEMBER 27, 2023

Epigenetics, often referred to as the ‘software’ of gene regulation, encompasses chemical modifications to DNA, such as methylation, and modifications of other proteins that organise DNA in the nucleus, known as histones, as well as RNA.

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DNA Regulations Disease RNA 289

Science Daily: Pharmacology News

MAY 29, 2024

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. Clarifying these molecular mechanisms contributes greatly to designing gene-editing tools accurate enough for gene therapy treatments.

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RNA DNA Therapies Treatment 97

Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

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RNA DNA Clinical Trials Therapies 130

Drug Discovery World

MAY 26, 2023

The top stories: Biopharma investment drops by nearly 50% in 2022 Cell therapy, RNA technology, antibody drug conjugates and AI/machine learning are among the key areas of investment for pharma, according to a new report. investment to target cancer and neurological diseases PharmEnable has closed a pre-series A investment round of $7.5

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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Therapies Clinical Trials Vaccine Disease 130

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

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Small Molecule Pharmacy RNA Packaging 246

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

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Science RNA Disease Protein Expression 147

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Read the article – Three trends in the antibody-drug conjugate (ADC) market Expert view: What’s next for cell and gene therapy? Read the article – Expert view: What’s next for cell and gene therapy?

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FDA Approval Therapies Small Molecule Clinical Trials 147

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

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Therapies RNA Licensing Licensing 40

Fierce BioTech

NOVEMBER 16, 2023

Beyond cfDNA: Harnessing the Power of Exosomes to Improve Sensitivity for Cancer Mutation Detection Liquid biopsy markers are advantageous because they can be used to assess overall prognosis, response to therapy, and likelihood of relapse or progression. Download now. Download now.

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RNA DNA Therapies Treatment 52

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

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FDA Approval Treatment FDA DNA 75

Drug Discovery World

APRIL 11, 2023

The molecule, known as lissodendoric acid A, appears to counteract other molecules that can damage DNA, RNA and proteins and even destroy whole cells. Dysregulation of mitophagy is closely linked to the development of several diseases, with USP30 modulation offering a potential treatment.

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Drug Research Research Drugs RNA 130

Drug Discovery World

MAY 23, 2024

Reece Armstrong speaks to Samuel Deutsch , Chief Scientific Officer of Nutcracker Therapeutics about the RNA landscape and how it can benefit the development of immunotherapies. RA: What role does RNA play in the development of effective immunotherapies? SD: This is definitely a challenge.

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RNA Therapies Engineering Disease 147

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

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

Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

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RNA Small Molecule Vaccine FDA Approval 147

Drug Target Review

NOVEMBER 13, 2023

The instructions to form a cell are encoded in DNA strands, wrapped in proteins and RNA, and wound together into a 3D structure called chromatin. Another finding was that regulatory T cells had distinct chromatin architecture features called DNA loops. This indicated other Foxp3 protein-containing complexes may be involved.

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Government DNA Regulations RNA 104

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43

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Therapies RNA Vaccine Small Molecule 130

Drug Discovery World

APRIL 16, 2023

The randomised KEYNOTE-942 trial assessed the efficacy of mRNA-4157/V940 in prolonging RFS in patients with resected, stages IIIB/IIIC/IIID and IV melanoma when given in combination with pembrolizumab, the standard-of-care adjuvant therapy in this patient population. in the combination arm and 62.2%

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Vaccine Immune Response Medical Schools Trials 130

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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Small Molecule Clinical Trials Drugs Therapies 84

Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. For both cancer and healthy genomes, we find that cysteine acquisition is a ubiquitous consequence of genetic variation that is further elevated in the context of decreased DNA repair.

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Small Molecule RNA DNA Therapies 52

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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Drugs Science Therapies Targeted Protein Degradation 189

Drug Target Review

SEPTEMBER 4, 2023

I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. 7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria.

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Drugs RNA DNA Disease 111

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines.

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RNA DNA Disease Therapies 52

Drug Target Review

APRIL 9, 2024

In a previous study, we showed that these peptides decorate all cellular RNAs and thus impair the binding of RNA-binding proteins to RNA. For his postdoctoral stay he joined the group of André Nussenzweig, where he started to work on DNA repair, particularly focusing on the role of histone H2AX.

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RNA Cell Biology Clinical Development DNA 64

The Open Targets Blog

AUGUST 15, 2023

Research in mice has shown that RBM3 expression is protective against misfolded protein damage, suggesting the protein as an interesting target in the development of therapies for neurodegenerative conditions such as Alzheimer’s disease. Curiously, these followed a theme in molecular function: the regulation of splicing*.

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RNA Treatment Regulations DNA 52

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. DNA vectors compared to standard linear mRNA-based expression. “We

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RNA DNA Therapies Disease 130

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. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

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Therapies Disease Medical Schools Virus 52

The Pharma Data

MARCH 7, 2022

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. Cas9 binds and uses RNA as an address-tag. The AI-designed blocking protein was the cargo of the dCas9-RNA construct.

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RNA DNA Packaging Small Molecule 52

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. Arrakis is the capstone of my career.

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Small Molecule RNA Clinical Trials DNA 52

Drug Discovery World

AUGUST 10, 2022

But decades of research led to improvements in cancer therapy, centred around the idea that one’s immune system could aid in treatment. Next, the advent of antibody treatments in 1997 created the first targeted cancer therapy 2. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic.

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