Drug Discovery World

JULY 15, 2024

Sapetschnig said that STORM will continue to develop a pipeline of therapies inhibiting RNA modifying enzymes and advance this candidate towards clinical development. From 5-10 April 2024 in San Diego, the cancer research community gathered for the annual meeting of the American Association for Cancer Research (AACR).

Research 52

Research In-Vivo Antibody RNA 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. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1

RNA 52

RNA In-Vivo Gene Therapy Genetic Disease 52

XTalks

AUGUST 12, 2020

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).

RNA 52

RNA Protein DNA In-Vivo 52

XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

Gene Expression 112

Gene Expression Development RNA Drugs 112

pharmaphorum

JANUARY 17, 2023

As it stands, mRNA vaccines and therapeutics can only provide benefits if they can reach the living cells and be processed into protein. The intracellular barriers include endosomal escape, RNA sensors, and endonucleases. The intracellular barriers include endosomal escape, RNA sensors, and endonucleases.

Vaccination 79

Vaccination Vaccine In-Vivo In-Vitro 79

pharmaphorum

JUNE 28, 2021

So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 90

In-Vivo Gene Editing Trials Drugs 90

Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT.

RNA 52

RNA In-Vivo Genotype Genetics 52

Drug Discovery World

AUGUST 3, 2022

Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake. Over the years, a range of different CPPs have been developed, including natural translocating proteins through to newly-designed computer-prediction sequences 3.

Bioavailability 52

Bioavailability In-Vivo RNA In-Vitro 52

The Pharma Data

DECEMBER 10, 2020

The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response. The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response.

Vaccination 52

Vaccination Vaccine Immune Response Protein 52

Drug Discovery World

SEPTEMBER 28, 2022

Artificial intelligence (AI) is notorious for its industry hype, touted to revolutionise the way multiple sectors operate over the next five to 10 years. Its application in pharma, most commonly in drug discovery, has been steadily growing with global partnerships between tech vendors and pharma companies becoming more commonplace.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

Protein 59

Protein Antibody Engineer Scientist 59

Drug Discovery World

SEPTEMBER 6, 2022

This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate. These genetic switches assist transcription factors in binding to the promoter region.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

APRIL 12, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

JANUARY 20, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

NOVEMBER 30, 2023

This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It The next presentation attended in this track was the keynote presentation, which was given by Kate Smith, PhD, Head of UK Protein & Cell Sciences at GSK. GSK has developed high-throughput mammalian and E.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Drug Discovery World

DECEMBER 15, 2022

Potential solutions: For CRISPR systems, the likelihood of off-target editing has been shown to be influenced by a variety of factors, ranging from the design of the gRNA 1 and Cas9 protein 2 , to the targeted cell type 3 , to name a few examples. In vivo approaches involve delivering the gene editing components directly into the patient.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

NOVEMBER 15, 2022

Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. . It has made its way into almost every aspect of drug discovery, whether it’s exploring basic biology, modelling diseases, identifying and validating targets, or performing drug screens. .

DNA 98

DNA Drugs Antibody Engineer 98

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 52

XTalks

APRIL 22, 2022

Flow cytometry is an analytical technique that is used to detect the presence of cell surface markers as well as intracellular proteins using antibodies in individual cells suspended in a buffer solution, which pass through a laser beam one at a time ( Figure 1 ). What is Flow Cytometry?

Clinical Trials 98

Clinical Trials Clinical Trials Trials Antibody 98

Drug Discovery World

JANUARY 17, 2024

Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer. As such there is a need to develop fast and accurate methods to assess drug safety and efficacy, facilitating early success or failure.

Medicine 59

Medicine DNA Genome Genomics 59

Drug Discovery World

OCTOBER 13, 2022

Prior to the pandemic, messenger RNA (mRNA) drug development was niche and predominantly pointed at different disease targets, including infectious diseases and cancers. One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. Why mRNA has potential . The rise of global demand .

Drugs 52

Drugs Manufacturing Vaccination Vaccine 52

The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

Drug Discovery World

JANUARY 23, 2023

This circumvents several technical challenges inherent to the genetic manipulation of differentiated cells in ex vivo culture or intact tissues. In cell therapy, regenerative and adoptive immunotherapies continue to bridge the gap between genetic engineering strategies and their clinical application.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

OCTOBER 20, 2022

used conventional flow cytometry analysis and FACS (fluorescence-activated cell sorting) to validate a pan-CAF marker along with CAF subpopulation-specific markers identified using single-cell RNA sequencing 3. Fibroblasts in the tumour microenvironment. This is of particular importance in dense tissues like breast and pancreas. Elyada et al.

Antibody 52

Antibody In-Vivo Life Science Development 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59