XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

Gene Therapy 105

Gene Therapy Gene Genome Genomics 105

Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers. The post Partners working to realise the future of genomic medicine appeared first on Drug Discovery World (DDW).

Genome 52

Genome Genomics Gene Editing Medicine 52

Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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Gene Therapy Gene Life Science Bioinformatics 52

Drug Discovery World

DECEMBER 5, 2022

Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. . In the study, researchers used ‘fossilised’ virus DNA sequences contained within animal genomes to reconstruct the long-term evolutionary history of parvoviruses.

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Gene Therapy Gene Genetic Disease Genetics 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.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

pharmaphorum

OCTOBER 12, 2022

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

Genome 105

Genome Genomics Medicine Development 105

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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Gene Editing DNA Gene Gene Therapy 235

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.

Gene Therapy 52

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.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 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. bluebird bio’s Philip Gregory.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Gene Therapy Gene Gene Editing In-Vivo 40

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.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna Genomics general manager and chief scientific officer Eric Huang said: “Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA.”

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

Gene Therapy 59

Gene Therapy Gene Antibody FDA Approval 59

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

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Gene Editing Gene Gene Therapy In-Vivo 145

Drug Discovery World

AUGUST 15, 2023

The post New whitepaper explores digital PCR for lentiviral vector characterisation appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy In-Vivo Gene DNA 52

Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

Development 52

Development Gene Therapy RNA DNA 52

Drug Discovery World

MAY 16, 2023

New South African company NewBiologix has launched with the aim of developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies. Current gene and cell therapies primarily focus on rare diseases.

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Production Gene Therapy Gene Bioinformatics 52

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

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.

Gene Therapy 69

Gene Therapy Gene Clinical Trials Clinical Trials 69

Drug Discovery World

AUGUST 15, 2023

Download this new Whitepaper to learn about: Use of ViralSEQ Lentivirus Physical and Proviral DNA Titer Kits with the QuantStudio Absolute QDigital PCR System Measurement of lentiviral vector titers and provirus copies in transduced cells by digital PCR Long terminal repeat (LTR)-based quantitation enabling correlation of total (genomic) and infectious (..)

In-Vivo 52

In-Vivo DNA Genome Genomics 52

Pharmaceutical Technology

FEBRUARY 3, 2023

The field of genomic medicine has reached a true turning point. With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

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Genome Genomics Medicine Gene Therapy 244

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

Roots Analysis

FEBRUARY 23, 2022

Over the past few years, several companies have started offering a diverse range of genome sequencing products and services using various second and third generation sequencing technologies. Currently, close to 60 industry players are actively offering NGS kits for preparing DNA / RNA libraries. Players Engaged in the Domain.

Genetics 52

Genetics DNA RNA Reagent 52

Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

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Gene Therapy Gene Marketing Manufacturing 130

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. billion in funding. This is reflected by our recent share price performance outperforming the index in the past year.”

Drugs 75

Drugs Life Science Gene Therapy Genome 75

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Gene Therapy Gene Gene Editing DNA 98

XTalks

DECEMBER 14, 2022

Developed by MIT researchers Jonathan Gootenberg and Omar Abudayyeh, PASTE (Programmable Addition via Site-specific Targeting Elements) gene editing technology can insert genes as long as 36,000 DNA base pairs to liver cells in mice as well as several types of human cells.

Gene Editing 98

Gene Editing DNA Gene Gene Sequencing 98

XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

Life Science 105

Life Science Gene Therapy DNA Vaccination 105

Cloudbyz

JUNE 26, 2023

Patient Risk Stratification: Generative AI techniques can analyze patient data, including electronic health records (EHRs) and genomic information, to identify individuals at higher risk for certain diseases or adverse events. This can lead to shorter trial durations, reduced costs, and more accurate evaluation of treatment efficacy.

Clinical Research 74

Clinical Research Research Clinical Trials Clinical Trials 74

Cloudbyz

MARCH 6, 2024

Unlike traditional small molecule drugs, which are chemically synthesized, biologics are produced through complex biotechnological processes, often involving recombinant DNA technology. This allows for the creation of highly specific and targeted therapies that can modulate biological pathways with remarkable precision.

Gene 71

Gene Immune Response Manufacturing Gene Therapy 71

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. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

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Gene Gene Therapy Genetics DNA 262

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.

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Scienmag

JANUARY 27, 2021

— Researchers used single-molecule imaging to compare the genome-editing tools CRISPR-Cas9 and TALEN. Their experiments revealed that TALEN is up to five times more efficient than CRISPR-Cas9 in parts of the genome, called heterochromatin, that are densely packed. Credit: Composite photo by L. Brian Stauffer CHAMPAIGN, Ill.

Genome 52

Genome Genomics DNA Research 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein.

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Vaccination Vaccine DNA Protein 52

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52