Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. This allows for the selection of patients who are most likely to respond to a particular therapy, optimizing treatment efficacy and minimizing adverse effects.

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Gene Therapy Gene Medicine In-Vitro 52

pharmaphorum

JUNE 15, 2021

When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category. . The post Biogen’s ambitions in gene therapy hit by another failed pivotal trial appeared first on.

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Gene Therapy Gene Trials Genetics 105

BioSpace

MAY 10, 2021

Biogen and Capsigen forged a strategic collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to become transformative gene therapies that treat underlying genetic causes of various central nervous system and neuromuscular disorders.

Gene Therapy 52

Gene Therapy Gene Engineer Genetics 52

STAT News

JULY 11, 2022

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

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Gene Gene Therapy Genetic Engineering Engineer 111

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

STAT News

AUGUST 18, 2022

Food and Drug Administration approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its manufacturer, Bluebird Bio , STAT tells us.

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Gene Therapy Gene FDA Approval Pharmacy 98

Pharmaceutical Technology

APRIL 20, 2023

Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. The circVec platform is a modular genetic cassette that provides instructions for the generation of multifunctional circRNAs. Topic sponsors are not involved in the creation of editorial content.

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Gene Therapy RNA Gene Development 240

Scienmag

DECEMBER 3, 2020

A new study has found that a novel T cell genetically engineered by University of Arizona Health Sciences researchers is able to target and attack pathogenic T cells that cause Type 1 diabetes, which could lead to new immunotherapy treatments.

Genetic Engineering 40

Genetic Engineering Engineer Genetics Bacteria 40

Scienmag

JUNE 9, 2021

Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

Gene Therapy 53

Gene Therapy Gene Scientist Medicine 53

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

Scienmag

MAY 11, 2021

Proof-of-principle research shows that genes can be accurately edited in cells throughout the body Credit: Zhiqian Li Researchers at the University of California San Diego have laid the groundwork for a potential new type of gene therapy using novel CRISPR-based techniques.

Gene Therapy 63

Gene Therapy Genetics Gene Research 63

Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Pharmaceutical Technology

AUGUST 17, 2022

Biopharmaceutical contract development and manufacturing organisation (CDMO) AGC Biologics has entered a strategic collaboration with RoosterBio to expedite the manufacturing of cell and exosome therapies. These capabilities comprise cell and exosome genetic engineering for expressing therapeutic targets.

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Manufacturing Gene Therapy Genetic Engineering Gene 130

Pharmaceutical Technology

AUGUST 25, 2022

The development and usage of innovative technologies, as well as expertise from the selection of cells and genetic modification to intelligent automation of production and information technology systems, will be part of the centre. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Manufacturing 130

Manufacturing Engineer Gene Therapy Production 130

Scienmag

MARCH 1, 2021

The in-person Cell Culture Engineering XVII Conference has been postponed until October 16 – October 22, 2021 in Tucson, Arizona. To highlight the key role that the CCE community plays to free people worldwide from the shackles of the COVID-19 pandemic, we are planning two webinars during the original dates of the conference.

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Engineer Gene Therapy Gene Genetics 43

BioTech 365

AUGUST 11, 2020

FDA grants Orphan Drug Designation to Neurogene’s adeno-associated virus vector with engineered transgene encoding the human CLN7 gene NEW YORK–(BUSINESS WIRE)–Neurogene Inc.,

Gene Therapy 52

Gene Therapy Gene Engineer Drugs 52

XTalks

SEPTEMBER 27, 2023

Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. Scientists working at Poseida Therapeutics.

Life Science 59

Life Science Genetic Engineering Engineer Gene Therapy 59

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

pharmaphorum

JANUARY 29, 2021

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. Macrae explains. “So

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Advarra

JULY 6, 2023

When conducting a trial involving engineered genetic materials, incidents involving occupational safety and environmental protection are also reportable to the IBC. The IBC will review the incident in a convened committee meeting to determine if any additional actions are required.

Genetics 52

Genetics Engineer Research Gene Therapy 52

The Pharma Data

JANUARY 18, 2021

This disease is a debilitating form of genetic epilepsy in children that significantly impacts movement, motor control, cognition and quality of life, and there remains a need to alter the course of this disease early in life.”. “As About Taysha Gene Therapies. Assistant Professor in Pediatric Neurology at UT Southwestern.

Gene Therapy 52

Gene Therapy Gene Drugs Production 52

Scienmag

MAY 19, 2021

Grew over $200 billion since 2010, outlines new areas of promising application WASHINGTON, DC (May 19, 2021) – Human genetics and genomics contributed $265 billion to the U.S.

Genome 43

Genome Genomics Genetics Gene Therapy 43

Pharmaceutical Technology

JUNE 29, 2022

For cell and gene therapy applications, you need a variety of speciality enzymes of the highest purity, specificity, and consistency. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior. And it’s within the family – Novozymes!”.

Drugs 295

Drugs Gene Therapy Production Manufacturing 295

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million.

Gene Editing 64

Gene Editing Gene Genome Genomics 64

XTalks

JANUARY 26, 2024

This technology, which allows for precise editing of DNA at specific locations, has been a major focus in the field due to its potential to directly target and modify cancer-causing genes. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

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Research Gene Editing Gene Genetics 109

Cloudbyz

MARCH 6, 2024

The Advantages of Biologics: The rise of biologics has brought forth numerous advantages over traditional pharmaceutical therapies: Targeted Therapies: Biologics can be designed to target specific molecules or pathways implicated in disease pathology, leading to enhanced efficacy and reduced side effects compared to non-specific treatments.

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Gene Immune Response Manufacturing Gene Therapy 99

Scienmag

DECEMBER 28, 2020

Texas A&M AgriLife researchers’ work to aid mosquito control efforts To control mosquito populations and prevent them from transmitting diseases such as malaria, many researchers are pursuing strategies in mosquito genetic engineering.

Genetic Engineering 40

Genetic Engineering Genetics Gene Engineer 40

Scienmag

APRIL 27, 2021

Researchers have discovered new approach to cereal plant breeding that considers the internal calculator of plant seeds that makes them continuously reorganize themselves; the method has the potential to obsolete the world’s crops far more effectively Credit: UCPH FOOD The research was conducted at the Department of Food Science at the University (..)

Genetics 57

Genetics Research Gene Therapy Production 57

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB. percent) compared to the control group (16.3

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FDA Approval Gene Therapy Gene Trials 102

Pharmaceutical Technology

FEBRUARY 24, 2023

AbbVie is expanding its strategic collaboration with Capsida Biotherapeutics for the development of targeted genetic medicines to treat eye diseases with high unmet needs. In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner.”

Gene Therapy 130

Gene Therapy Gene Engineer Manufacturing 130

Scienmag

JANUARY 13, 2021

Study demonstrates the efficacy and therapeutic potential of select ionizable lipid nanoparticles for delivering mRNA to treat genetic diseases before birth Credit: Children’s Hospital of Philadelphia Philadelphia, January 13, 2021–Researchers at Children’s Hospital of Philadelphia and the School of Engineering and Applied Science (..)

Genetic Disease 52

Genetic Disease Research Engineer Genetics 52

Scienmag

JUNE 2, 2021

Researchers create novel CRISPR-based fly species as a new method of controlling gene drive spread Credit: Akbari lab, UC San Diego CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies.

Gene 98

Gene Engineer Development Research 98

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. In addition, Vir Biotechnology, Inc.

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FDA Approval Gene Therapy Gene Clinical Trials 110

Advarra

JANUARY 11, 2024

Personalized medicine using genetic resequencing techniques, such as cell and gene therapies, enables researchers to create more customized therapies for combatting cancer. Adoptive T Cell Therapies Adoptive T cell therapy is another form of cancer treatment leveraging the patient’s immune system.

Vaccination 52

Vaccination Vaccine Protein Antibody 52

NY Times

FEBRUARY 22, 2021

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.

Gene Therapy 54

Gene Therapy Trials Gene Research 54

Scienmag

MAY 28, 2021

Genetics toolkit targets less researched Culex mosquitoes, which transmit West Nile virus and avian malaria Credit: Gantz Lab, UC San Diego Since the onset of the CRISPR genetic editing revolution, scientists have been working to leverage the technology in the development of gene drives that target pathogen-spreading mosquitoes such as Anopheles and (..)

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Containment Genetics Research Gene 69

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

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Gene Therapy Gene Medicine Trials 98