Scienmag

JANUARY 28, 2021

— In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition. ROCHESTER, Minn.

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Gene Therapy Gene In-Vitro Clinical Research 74

Pharmaceutical Technology

MAY 4, 2023

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results. This cohort showed a safety profile identical to that found in older children and adults.

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In-Vitro Genetic Disease Medicine Genetics 246

Pharmaceutical Technology

APRIL 12, 2023

FoundationOne CDx is a sequencing-based in vitro diagnostic device that can identify alterations in 324 genes from tumour samples. The ROS1 gene is altered in approximately in 1-2% of lung cancer patients. Exposure to environmental factors can cause gene fusion which leads to upregulation of the ROS-1 enzyme.

Genome 130

Genome Genomics In-Vitro Gene 130

Roots Analysis

NOVEMBER 30, 2021

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies. How Players are Complying with Developing Market?

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Bioassay Gene Therapy Gene Development 52

Pharmaceutical Technology

MAY 12, 2023

Whilst the formulation had been successful in vitro , the oral administration in mice did not elicit activity. The latter partnership was recently expanded to focus on in vitro and in vivo delivery of mRNA after BioNTech pulled out of the deal with Matinas. Matinas announced the news in a May 10 statement.

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Vaccination Vaccine In-Vivo In-Vitro 130

Pharmaceutical Technology

APRIL 14, 2023

All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. Topic sponsors are not involved in the creation of editorial content.

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. MUNICH, Germany, Jan.

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Gene Therapy Gene Development In-Vivo 52

Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? Skills gap Bruce Levine, International Society for Cell & Gene Therapy (ISCT) Immediate Past President and Chair, Strategic Advisory Council, says a critical challenge that has become apparent for the CGT sector is the need for workforce development and training resources.

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Gene Therapy Gene Manufacturing Marketing 52

Scienmag

DECEMBER 10, 2020

For this, they successfully generated reconstituted chromatin bearing histone modifications in vitro. Credit: Nucleic Acids Research Scientists at Tokyo Institute of Technology decipher how to quantitatively assess the effects of specific epigenetic changes on the rate of transcription by developing a mathematical model.

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Gene Expression In-Vitro Gene Scientist 52

Drug Discovery World

MAY 1, 2023

Download Emulate’s Introduction to Organ-on-a-Chip Technology eBook to learn about: What Organ-Chips are, how they work, and why they are transforming the way drugs are discovered and developed How Organ-Chips overcome the main challenges of conventional in vivo and in vitro models The wide variety of insights and endpoint analyses Organ-on-a-Chip (..)

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In-Vivo In-Vitro Gene Therapy Gene 52

BioPharma Reporter

JUNE 29, 2021

The New York based company said the deal will expand its in vitro diagnostic, cell and gene therapy support capabilities.

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Reagent In-Vitro Gene Therapy Gene 52

Pharmaceutical Technology

MAY 24, 2023

It is building many MDSC-targeted, bi-functional ADCs for in vitro and in vivo characterisation, with lead selection expected by the end of next year. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Morphogenesis’ lead personalised cancer vaccine, IFx-Hu2.0,

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Gene Therapy In-Vivo In-Vitro Immune Response 130

Scienmag

MARCH 30, 2021

In vitro treatment of cells with these molecules reduced the expression of a gene that plays a key role in viral cell entry and a cytokine receptor.

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In-Vitro Gene Allergies Medicine 55

Drug Discovery World

MAY 1, 2023

The post New eBook: An introduction to organ-on-a-chip technology appeared first on Drug Discovery World (DDW).

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In-Vivo In-Vitro Gene Therapy Gene 52

Drug Discovery World

JUNE 30, 2023

Select mature stem cell lines can then be used in small-scale in vitro assays, and proven biochemical and phenotypic screens supporting target validation and deconvolution studies. Through the use of human induced pluripotent stem cells (iPSCs) derived cells, we can improve translation and reduce risk of clinical failure.

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Development Drugs In-Vitro Gene Therapy 52

pharmaphorum

DECEMBER 1, 2022

Exploring the implementation of complex in-vitro models for drug development. The pharmaceutical industry globally is realising the growing potential of in vitro tissue models for drug discovery, pathology modelling and validation, safety and toxicity. Billion by 2028. Billion by 2028. Event Hashtag: #3DCellCulture2023.

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BioTech 365

FEBRUARY 26, 2021

New Study Published in Cephalalgia Identifies Mechanistic Differences Among CGRP Migraine Treatments New Study Published in Cephalalgia Identifies Mechanistic Differences Among CGRP Migraine Treatments In vitro findings suggest specific classifications for calcitonin gene-related peptide (CGRP) pathway-based therapeutics based on mechanisms … (..)

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Drug Discovery World

AUGUST 22, 2023

In addition, total blood levels of sulforaphane (SFN) and SFN-metabolites were confirmed in the micromolar range, where efficacy is seen in vitro. Evgen Pharma has revealed the final data from its Phase Ib healthy volunteer study using the company’s new enteric coated tablet formation of its lead asset SFX-01.

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Gene Expression In-Vitro Gene Trials 52

Drug Discovery World

DECEMBER 13, 2022

One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. Co-author Hiroki Kurahashi added: “Patients with FCMD who carry the abnormal FKTN gene produce non-glycosylated α-DG, which makes them bedridden right from adolescence.

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RNA Genetics Gene In-Vitro 52

Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. We were excited to unveil data showing that two distinct chemical series exhibit METTL1 inhibition in vitro at low nanomolar concentrations with minimal interference with other RNA and protein methyltransferases.

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RNA In-Vivo In-Vitro DNA 62

FDA Law Blog

MARCH 31, 2024

Committee Chair Rodgers expressed her concerns that FDA regulation of LDTs would substantially increase the regulatory and financial burden on labs, causing them to stop offering many tests, such as those that are used in oncology and cell and gene therapies. This seems to have been the outcome that many lawmakers desired.

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Regulation In-Vitro Development Gene Therapy 64

Drug Discovery World

JANUARY 17, 2024

Likewise, human cells with a mutated MFN2 gene exhibited similar traits, as well as abnormalities in mitochondrial fission and lower energy (or ATP) production, indicating problems in the generation of ‘cellular energy’. The results were consistent with the in vitro experiments.

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In-Vitro Regulation Protein Gene 52

Drug Discovery World

FEBRUARY 6, 2024

Target identification and validation are where there is huge impact; simply put, genomics can be used to identify new drug targets by identifying genes that are overexpressed or mutated in diseased tissues. She speaks to Megan Thomas on how genomics can advance drug discovery and development.

Genome 57

Genome Genomics Genetics In-Vitro 57

pharmaphorum

FEBRUARY 19, 2021

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS.

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Gene Editing In-Vivo DNA In-Vitro 105

Bioengineer

JANUARY 16, 2024

In patients with T790M mutant NSCLC receiving osimertinib, 63% experienced loss of T790M at progression, commonly linked to histological transdifferentiation, KRAS mutations, or gene fusions. The presence of the T790M mutation during first or second-generation EGFR-TKI treatments is observed in 50-60% of patients. months vs. 15.2

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Genome Genomics Protein In-Vitro 119

Drug Discovery World

APRIL 3, 2023

As the industry moves to reduce the use of animals in research and testing, aptamers also support here, as they are discovered, developed, and manufactured entirely in vitro , removing the need for animals in antibody discovery processes. DDW’s Diana Spencer caught up with co-founder and CEO Dr Arron Tolley.

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Antibody Gene Therapy Gene In-Vitro 52

Drug Discovery World

JULY 3, 2023

This was centred around pre-clinical models (in vitro and in vivo) to support discovery and development, re-entering the fray with costimulatory agonists, and clinical development strategies for combinations with immune cell engagers​.

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Antibody In-Vitro Engineer In-Vivo 52

Drug Discovery World

JULY 10, 2023

For projects that utilise gene targeting in ESCs, founders are chimeric (composed of genetically distinct cells derived from two different sources: modified ESCs and a host blastocyst). Yet when seeking to obtain an existing model, researchers often encounter obstacles related to intellectual property, availability, or licensing.

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Genetics Gene Editing Genetic Engineering In-Vitro 52

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

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Gene Therapy Drugs Gene Manufacturing 52

Roots Analysis

JANUARY 14, 2024

Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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Contract Manufacturing Manufacturing Protein Bacteria 40

pharmaphorum

NOVEMBER 1, 2022

In 2023 we will see the return of SAE’s RNA Therapeutics conference to a live in-person event, showcasing an update on mRNA therapeutic applications, coupled with an industry outlook of the field. We will also explore the latest trends in novel RNA therapies, including developments in AI modelling and small molecule modulation of RNA.

RNA 52

RNA Genetics Gene Editing In-Vitro 52

Drug Discovery World

OCTOBER 27, 2023

Organ-on-a-chip technology allows researchers to recreate the human tumour microenvironment in vitro, using human cells and capturing much of the complexity that characterises in vivo environments,” she says. But can organ-chips entirely replace other models? Not yet, according to Ewart. While the FDA’s Modernisation Act 2.0

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Research Gene Therapy In-Vivo In-Vitro 70

Drug Discovery World

JANUARY 12, 2023

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . What are organoids? .

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Gene Editing Gene Drugs In-Vivo 98

STAT News

FEBRUARY 23, 2023

Ovarian cancer has long been one of the deadliest cancers to strike women. No tests are available to detect it early, so most patients are diagnosed in later stages, when the cancer is more likely to have spread. Fewer than half of women survive 5 years past their diagnosis, and 80 percent have no known risk factors.

Genetics 111

Genetics In-Vitro Research Gene 111

XTalks

APRIL 6, 2022

Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. The personalized ctDNA blood test uses the RaDaR™ assay, a liquid biopsy platform developed by the company Inivata. Photo source: Inivata. Around 85 percent of cases are non-small cell lung cancer (NSCLC).

DNA 105

DNA In-Vitro Doctors Doctor 105

Roots Analysis

OCTOBER 16, 2023

DNA and Gene Cloning involves the isolation of a DNA sequence of any species ( often a gene ) and its insertion into a vector to enable growth without any alteration in the original DNA sequence. These clones can be manipulated and mutated in vitro to alter the expression and function of a protein.

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DNA Gene Protein In-Vitro 40

The Pharma Data

DECEMBER 20, 2020

On May 14, the company presented data from the vibegron EMPOWUR 52-week extension study and data by age groups from the 12-week placebo-controlled EMPOUR study at the 2020 American Urological Association Annual Meeting. The drug is a once-daily, beta-3 adrenergic agonist. David Staskin, principal investigator of EMPOWUR and a urologist with St.

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In-Vitro FDA Approval Manufacturing Drugs 52