Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. .

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Gene Therapy Gene Genetic Disease Genetics 52

Roots Analysis

JUNE 1, 2022

DNA cloning is the field of synthetic biology which can be helpful to understand the effect of mutation on a particular gene. Many diseases that can be treated using DNA cloning, such as leukemia and sickle cell anemia, involving the replacement of defected gene. DNA Cloning Kit Providers – Current Market Landscape.

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DNA Genetics Reagent Marketing 52

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

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Drug Discovery World

APRIL 24, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. This webinar will detail how Oxford Nanopore evaluated long-read nanopore sequencing as a comprehensive and sensitive QC method for rAAVs which is supporting internal research activities at AstraZeneca.

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Genome Genomics DNA Gene Therapy 52

Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate.

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Gene Therapy Gene Genome Genomics 52

Drug Discovery World

APRIL 16, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. This webinar will highlight how long-read nanopore sequencing as a comprehensive and sensitive QC method for rAAVs were evaluated — which is supporting internal research activities at AstraZeneca.

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Packaging Packaging Genome Genomics 52

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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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

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Gene Therapy Gene Genome Genomics 52

Drug Discovery World

APRIL 17, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. This webinar will detail how Oxford Nanopore evaluated long-read nanopore sequencing as a comprehensive and sensitive QC method for rAAVs which is supporting internal research activities at AstraZeneca.

DNA 52

DNA Genome Genomics Gene Therapy 52

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Shares in bluebird ticked up following the announcement.

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Gene Therapy Gene Trials Gene Expression 59

Roots Analysis

FEBRUARY 27, 2024

Over the last few decades, various viral vector manufacturing have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option.

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Manufacturing Gene Therapy Gene DNA 52

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Engineer Genetics Clinical Research Research 52

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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DNA Gene Gene Editing Scientist 56

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. Food and Drug Administration.

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Gene Therapy Gene Gene Editing Genetics 52

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drugs Gene Editing Genome Genomics 52

Pharmaceutical Technology

JUNE 4, 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. bluebird bio is also developing drugs based on DNA and retro-virus vectors.

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Gene Therapy DNA Gene Genetics 246

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. ” The Institute will be headquartered in 334,000 sq.

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Genetics Medicine RNA Scientist 52

Delveinsight

FEBRUARY 18, 2021

Bluebird suspends gene therapy trials after two sickle cell patients develop cancer. Bluebird bio has stopped two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Cortexyme tripped up by FDA hold over liver toxicity.

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Gene Therapy Gene Trials Clinical Trials 52

Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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Gene Expression Regulation Gene DNA 97

Roots Analysis

FEBRUARY 23, 2022

Further, NGS library preparation kits offer several benefits, such as low requirement of starting material, generation of longer RNA / DNA sequences and production of superior quality nucleotide libraries, thereby, catering to existing unmet needs of the research domain. Next Generation Sequencing (NGS) Library Preparation Kits. Web: [link].

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Genetics DNA RNA Reagent 52

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

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Gene Therapy Marketing Gene DNA 52

Scienmag

APRIL 15, 2021

Research provides guidance for global application Detecting hidden genetic defects by applying an existing method to an existing datasets.

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Genetics DNA Research Gene Therapy 41

Pharma Packaging Solutions

JUNE 30, 2023

Semi-Synthesized: Though the term “biologic” is accurate for the extracted materials listed above, it is more often used to describe therapeutics that are produced with recombinant DNA technology. As biopharma technology progresses, becoming both more prevalent and available, the need for high-quality biopharma packaging increases.

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Recombinant DNA Technology Packaging Packaging Gene Therapy 52

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . We used these peptides to act as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye.” .

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Gene Editing Gene Gene Therapy Genetics 98

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Editing Gene In-Vivo Gene Therapy 147

Scienmag

APRIL 15, 2021

High resolution electron microscopy imaging shows how damage-sensing proteins recognize DNA breaks and then bridge them together Credit: He Lab From cancer treatment to sunlight, radiation and toxins can severely damage DNA in both harmful and healthy cells.

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DNA Protein Gene Therapy Gene 46

XTalks

DECEMBER 4, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

Scienmag

FEBRUARY 11, 2021

Study in The CRISPR Journal reports the app advances CRISPR gene therapy R&D; 18-year-old software savant helped lead the app’s development Wilmington, DE, Feb. The app reveals potentially risky DNA […].

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Gene Gene Therapy DNA Scientist 73

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. Eventually, they will need assistance with breathing.

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Gene Therapy Gene FDA Approval DNA 98

pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. First of all it will be used for ultra-rare monogenic disease.

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Gene Editing Gene Gene Therapy In-Vivo 145

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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Research Gene Editing Gene Genetics 118

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

APRIL 17, 2024

As for gene-modulating approaches, I want to point out that our therapeutic candidates do not in any way modulate the gene itself. Increasing gene expression via protein replacement or gene therapy has been shown to have limitations and drawbacks. Can you give some examples?

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Gene Expression Gene Genetic Disease RNA 59

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

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Gene Therapy Gene Life Science DNA 111

Delveinsight

DECEMBER 17, 2020

Following that hypothesis, scientists designed oral inhibitors, which could target mitochondrial DNA (mtDNA). Instead of directly targeting existing mitochondria, the team went after mitochondrial DNA transcription that is vital to the formation of new mitochondria and the production of energy. million in financing.

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