Drug Discovery World

AUGUST 15, 2023

Recombinant lentiviral vectors are used for ex vivo transgene delivery in several gene-modified cell therapies, such as chimeric antigen receptor (CAR) T cell therapy.

In-Vivo 52

In-Vivo DNA Genome Genomics 52

Drug Discovery World

AUGUST 15, 2023

Reliable analytical methods such as digital PCR to characterise lentiviral vectors and the cells transduced with them are critical to helping ensure the safety and efficacy of these biologics.

Gene Therapy 52

Gene Therapy In-Vivo Gene DNA 52

BioTech 365

SEPTEMBER 2, 2020

(Nasdaq: SRNE, “Sorrento”) and SmartPharm Therapeutics, Inc. (“SmartPharm”) announced today that Sorrento has completed the acquisition of SmartPharm, a gene-encoded protein therapeutics company developing non-viral DNA and … Continue reading →

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DNA In-Vivo Protein Gene 40

Pharmaceutical Technology

MARCH 24, 2023

Under the terms of the deal, Moderna has purchased an option to license ctLNP and closed-ended DNA (ceDNA) technology from Generation Bio. Both companies will use the partnership for advancing in vivo immune cell targeting as a new class of genetic medicines.

Genetics 130

Genetics Medicine Development In-Vivo 130

Pharmaceutical Technology

FEBRUARY 14, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. These systems include human and mouse cell lines, and even in vivo in live animals.

In-Vivo 130

In-Vivo DNA Antibody Gene 130

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

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. billion, compared to $19.9 Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. In vivo, METTL1 inhibitors induce tumour growth inhibition in both immune-deficient and immune-competent mouse strains. We were there to present the latest findings from our research.

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RNA In-Vivo In-Vitro DNA 64

Pharmaceutical Technology

MARCH 9, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. However, not all innovations are equal and nor do they follow a constant upward trend.

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In-Vivo Antibody Genetics Gene 130

pharmaphorum

FEBRUARY 19, 2021

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. Khalili is a named inventor on patents that cover the viral gene editing technology and also holds equity in Excision, where he will act as chief scientific consultant.

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Gene Editing In-Vivo DNA In-Vitro 105

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

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.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

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.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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

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

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

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

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Life Science Gene Editing Development Clinical Trials 119

XTalks

AUGUST 12, 2020

Despite the effectiveness of anti-retroviral therapy (ART) in significantly improving health, quality of life and reducing mortality among HIV-positive individuals, new research shows that persistent infections may be due to the ability of the virus to take refuge in distinct subsets of immune T cells.

RNA 52

RNA Protein DNA In-Vivo 52

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. .

DNA 98

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.

Gene Editing 98

Gene Editing Trials Drugs Gene 98

Roots Analysis

MARCH 1, 2023

Disruption of the integrity and function of DNA at the gene or chromosomal level can lead to heritable mutations, resulting in genetic disorders, birth abnormalities, or cancer. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

Genotoxicity 52

Genotoxicity DNA Genetics Bacteria 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

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.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 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

Drug Discovery World

NOVEMBER 9, 2023

Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’. 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.

Engineer 52

Engineer Antibody Protein In-Vivo 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

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.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

Roots Analysis

AUGUST 8, 2023

Furthermore, a wide variety of drugs and macromolecules, including DNA , proteins, and imaging agents, can be encapsulated in liposomal vesicles due to their unique ability to entrap both lipophilic and hydrophilic substances. Bangham, in 1961 at the Babraham Institute, University of Cambridge.

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Drug Delivery Systems Drug Delivery Drugs In-Vivo 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.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry.

Engineer 52

Engineer Containment In-Vivo Production 52

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