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

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RNA In-Vivo Gene Therapy Genetic Disease 52

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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Gene Gene Therapy Gene Expression Regulation 40

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).

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RNA Protein DNA In-Vivo 52

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

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DNA Genome Genomics RNA 98

Drug Discovery World

NOVEMBER 15, 2022

Rapid and accurate DNA synthesis . Conventional DNA synthesis, typically used for making sequences such as primers, employs phosphoramidite chemistry, a method that was developed in the 1980s and is still considered the gold standard for DNA synthesis. .

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DNA Drugs Antibody Engineer 98

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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Gene Editing Trials Drugs Gene 98

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

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Gene Editing Gene Engineer DNA 52

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

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Photo courtesy of Science Advances.

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Gene Editing DNA Genome Genomics 52

Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

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Antibody Protein Engineer In-Vivo 59

Drug Discovery World

NOVEMBER 13, 2023

Noel Pauli, PhD, Group Leader, Antibody Engineering, Adimab, on: ‘Harnessing in vivo diversities with a yeast-based platform for the discovery of antibodies against multi-pass membrane proteins’. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

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Protein Antibody Engineer Scientist 59