Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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Gene Expression Gene Packaging Packaging 52

Scienmag

JULY 12, 2021

The function of a protein can depend on its abundance in a cell. So, when investigating the properties of a new protein, it is essential to make sure that the same amount is produced by every cell. Researchers at Baylor College of Medicine and Rice University have found a new way to do just that […].

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Gene Expression Engineer Gene Protein 63

Scienmag

NOVEMBER 11, 2020

Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […].

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Drug Discovery World

NOVEMBER 20, 2023

Engineering biology is reshaping the world, from enabling the rapid development of vaccines to providing alternatives to petroleum fuels. Central to engineering biology is the design – or redesign – and assembly of biological components to generate a desired function.

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Drug Discovery World

MAY 29, 2023

The US Food and Drug Administration has approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a genetic disorder that affects the connective tissue in the skin and nails and results from mutation(s) in the COL7A1 gene.

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Gene Therapy FDA Approval Gene Genetics 52

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

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Gene Therapy FDA Approval Gene Clinical Trials 95

Pharmaceutical Technology

APRIL 20, 2023

In addition to new regulatory functionalities, this feature may result in stronger and more durable protein expression. Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy RNA Gene Engineer 240

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. . Limitations of organoids.

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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 4, 2023

Originated by the company’s in-house discovery engine, ATX-01 is designed to target microRNA 23b (miR-23b), a pathogenic gene associated with regulating the muscleblind-like protein (MBNL) expression involved in DM1. It has a dual mechanism of action that targets toxic dystrophia myotonica protein kinase and MBNL protein.

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Clinical Trials Clinical Trials Trials Protein 130

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.

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Gene Editing Gene In-Vivo Genome 52

Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Editing Gene Manufacturing 162

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’.

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Protein Antibody Engineer Scientist 59

Drug Discovery World

NOVEMBER 27, 2023

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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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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Gene Therapy Gene Manufacturing Medicine 52

Pharmaceutical Technology

DECEMBER 8, 2022

Directed against a cell membrane protein called CD19, Yescarta uses the immune system of the patient to destroy cancer. It is created by taking T cells from the patient’s blood and then engineered in the laboratory to express chimeric antigen receptors. . By Cytiva Thematic.

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Licensing Licensing Gene Therapy Gene 305

Scienmag

DECEMBER 16, 2020

Surprisingly simple method could provide a new tool for producing specialized cytoplasm for reproductive medicine Credit: Kyushu University In a new study published in the journal Nature, researchers in Japan report that activating just eight genes for producing gene-controlling proteins is enough to convert mouse stem cells directly into oocyte-like (..)

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Gene Protein Medicine Research 52

Scienmag

MARCH 19, 2021

Treatment involving a single injection has long-lasting effects BOSTON – Researchers have used a genetic engineering strategy to dramatically reduce levels of tau–a key protein that accumulates and becomes tangled in the brain during the development of Alzheimer’s disease–in an animal model of the condition.

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Gene Therapy Genetic Engineering Gene Engineer 66

Drug Discovery World

NOVEMBER 9, 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. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

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Pharmaceutical Technology

JULY 6, 2022

These involve mRNA molecule engineering, including sequence modifications to enhance stability and boost protein expression, and mRNA vaccine formulations appropriate for SARS CoV-2 vaccines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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The Pharma Data

DECEMBER 22, 2020

SAITAMA, Japan–( BUSINESS WIRE )– After a capital increase in the initial closing of its series A round, with investments by Mitsubishi UFJ Capital and Real Tech Fund , Epsilon Molecular Engineering Inc. The bio industry has progressed to a point where it deals with proteins from an engineering perspective.

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Engineer Drugs Protein Antibody 52

XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

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Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. Specialty enzymes are proteins that can act as very specific biocatalysts to accelerate reactions and produce the desired target molecule in pharmaceuticals,” explains Kristoffer Laursen, Head of R&D at Novo Nordisk Pharmatech.

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Drug Discovery World

FEBRUARY 1, 2024

This paid-for advertorial by Twist Bioscience appeared in the Therapeutic Antibodies Guide – DDW Volume 25 – Issue 1, Winter 2023/2024 Improving gene synthesis speed can reduce workflow times, saving you invaluable time, says Twist Bioscience. But speedy gene synthesis is valuable well beyond antibody development.

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Antibody Gene Protein Manufacturing 64

The Pharma Data

JANUARY 17, 2021

The companies will evaluate ViGeneron’s proprietary, intravitreally injected vgAAV vectors for delivering a novel therapeutic protein to develop a gene therapy treatment for a highly prevalent eye disease. The second, REVeRT vector platform, targets diseases caused by mutations in large genes. MUNICH, Germany, Jan.

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Gene Therapy Gene Research Protein 52

Drug Discovery World

NOVEMBER 30, 2023

This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It coli expression systems and is using high-throughput expression pipelines to screen constructs for optimal expression to generate protein and cellular reagents. GSK has developed high-throughput mammalian and E.

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Antibody Protein Engineer In-Vivo 59

Drug Discovery World

MARCH 22, 2023

The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times greater T cell expansion in models, resulting in increased antitumour immune activity and durability. They also observed increased antitumour immune activity and longevity of the engineered T cells.

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Gene Editing Gene Engineer Regulation 52

Drug Discovery World

NOVEMBER 15, 2022

The brainchild of multidisciplinary experts in computer science, physics, engineering, and biology, the field of synthetic biology has not only rapidly progressed in the last few decades but it’s also becoming more accessible and cost-effective. The longer the DNA gets, the longer it will take for labs to receive it,” notes Dr Daniels.

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DNA Drugs Antibody Engineer 98

Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. One example: Zynteglo, a newly approved gene therapy priced at a record-breaking $2.8M It has taken a concerted effort to evolve cell and gene therapies into what they are today, and the technology is advancing faster than ever. Fierce Pharma.

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Gene Therapy Gene Manufacturing Development 52

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. The mRNA constructs used in COVID-19 vaccines, for example, direct cells to produce a version of the “spike” protein studding the surface of SARS-CoV-2.

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Vaccination Vaccine Protein Antibody 52

Drug Discovery World

MAY 23, 2024

Could you discuss the benefits of encoding for multiple proteins and the associated anti-cancer effects? RNA-based approaches allow the design of more complex molecules, as the proteins are freshly produced in the patients’ cells and tissues being delivered as mRNA nanoparticles with standard manufacturing and formulation processes.

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RNA Protein Manufacturing Production 59

Cloudbyz

MARCH 6, 2024

Understanding Biologics: Biologics are a class of therapeutic agents derived from living organisms, such as cells, tissues, or proteins. The Promise of Gene and Cell Therapies: Beyond monoclonal antibodies and protein-based biologics, the field of biotechnology is rapidly advancing towards the development of gene and cell therapies.

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Gene Immune Response Manufacturing Gene Therapy 75

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

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Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

JANUARY 17, 2024

The company aims to advance platform technologies including chemoproteomics, molecular glue and covalent drug discovery, high-throughput screening, protein and antibody engineering, gene and epigenetic editing, gene and cell therapies, drug delivery, and data science.

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Drugs Drug Delivery Life Science Gene 52

Drug Discovery World

OCTOBER 20, 2023

Its proprietary technology, Proximity Extension Assay (PEA), provides high throughput protein analysis. Through the purchase, Thermo Fisher will also gain a library of more than 5,300 validated protein biomarker targets. Olink specialises in solutions for proteomics discovery and development.

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Drugs Antibody DNA Gene Therapy 52

Drug Discovery World

OCTOBER 24, 2023

A BioIndustry Association (BIA) report lists the widespread adoption of artificial intelligence (AI) and machine learning (ML), the advancement of gene editing technologies, the power of engineering biology, and a growing interest from major pharmaceutical players as key trends driving the sector.

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Gene Editing Engineer Clinical Trials Clinical Trials 52

XTalks

OCTOBER 21, 2020

The answer may lie in the genetic code of the virus, which scientists at Duke University have found contains several silent mutations that affect protein folding. Related: Study Dispels Claim that COVID-19-Causing Coronavirus is Lab-Engineered. These mutations may thus be “silent” as they do not alter protein structure.

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