XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. How do Casgevy and Lyfgenia Work?

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

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pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. — Brad Loncar (@bradloncar) August 9, 2021.

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pharmaphorum

APRIL 22, 2022

Astellas has said it will book a $170 million impairment charge in its fourth quarter results as a result of a decision to halt the development of three gene therapy candidates for Duchenne muscular dystrophy in preclinical development. The post Astellas takes $170m charge as it drops DMD gene therapies appeared first on.

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pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6

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BioPharma Reporter

OCTOBER 20, 2022

Forge Biologics has added plasmid DNA manufacturing to its roster of services, enabling it to vertically integrate its production process.

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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.

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pharmaphorum

NOVEMBER 20, 2020

Just over a year after its first phase 3 trial of its Engensis gene therapy for painful diabetic peripheral neuropathy (DPN) bombed, Helixmith has started dosing patients in a new study. The South Korean biotech says the DNA plasmid-based therapy has been administered to a patient at Innovative Research of West Florida.

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Drug Discovery World

MAY 24, 2023

Touchlight, a company providing DNA services and manufacturing enzymes, has completed the redevelopment and expansion of its UK manufacturing facility in London, UK. The expanded facility’s manufacturing capacity has tripled and is now capable of producing more than 8kg a year.

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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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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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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. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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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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Drug Discovery World

NOVEMBER 23, 2022

One critical bottleneck in Cell and Gene Therapy (CGT) manufacturing is the production of high-quality plasmid DNA (pDNA), a vital building block for mRNA-based vaccines, viral vector-based cell or gene therapies that require pDNA as a starting material.

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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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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.

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? They note that the gene therapy market in general, and the lentiviruses market by association, is growing enormously. DDW’s Megan Thomas finds out.

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XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. XTALKS WEBINAR: Importance of Early Integrated Evidence Strategy for Cell & Gene Therapies. How Does the Gene Therapy Adstiladrin Work?

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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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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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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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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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Drug Discovery Today

FEBRUARY 24, 2022

Global cell and gene therapy development and manufacturing business expands into Bruntwood SciTech’s Alderley Park to boost manufacturing capacity.The 17,000 sq ft high-specification lab space is currently under construction, expanding plasmid DNA and viral vector capabilities in Europe.The facility will support therapeutic developers with rapid access (..)

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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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BioPharma Reporter

JANUARY 17, 2022

Andelyn Biosciences says the FDA's acceptance of its GMP plasmid DNA drug master file (DMF) enables the CDMO to vertically integrate its clientsâ manufacturing process, condensing timelines for developers to begin manufacturing to just three months.

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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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Pharmaceutical Technology

FEBRUARY 23, 2023

GenScript ProBio has announced a strategic collaboration with RVAC Medicines to manufacture GMP-grade plasmid DNA (pDNA) for the latter’s RVM-V001, an mRNA Covid-19 vaccine candidate. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery Today

OCTOBER 19, 2020

Former Lonza President of Biologics Manufacturing joins to continue rapid expansion of DNA contract manufacturing; Touchlight’s synthetic dbDNA in high demand as starting material in the manufacture of advanced therapies such as mRNA vaccines and cell and gene therapies

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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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BioTech 365

OCTOBER 28, 2020

DUBLIN–(BUSINESS WIRE)–The “Viral Vector and Plasmid DNA Manufacturing Market – Growth, Trends, and Forecasts (2020 – 2025)” report has been added to ResearchAndMarkets.com’s offering.

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Pharmaceutical Technology

MAY 17, 2023

In addition, the AAV and LVV platforms feature in-network plasmid DNA (pDNA) supplies, in-house capability for major testing and the use of cell factories and fixed bed bioreactor for adherent protocols along with wave and stirrer bioreactors for suspension. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

NOVEMBER 14, 2022

Don’t forget to register for this free webinar, hosted by DDW and supported by Catalent, Early phase support for plasmid DNA supply helps secure future manufacturing for advanced therapies.

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Drug Discovery World

NOVEMBER 7, 2022

One critical bottleneck in Cell and Gene Therapy (CGT) manufacturing is the production of high-quality plasmid DNA (pDNA), a vital building block for mRNA-based vaccines, viral vector-based cell, or gene therapies that require pDNA as a starting material. .

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Drug Discovery World

NOVEMBER 4, 2022

One critical bottleneck in Cell and Gene Therapy (CGT) manufacturing is the production of high-quality plasmid DNA (pDNA), a vital building block for mRNA-based vaccines, viral vector-based cell, or gene therapies that require pDNA as a starting material. .

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Drug Discovery World

OCTOBER 31, 2022

One critical bottleneck in Cell and Gene Therapy (CGT) manufacturing is the production of high-quality plasmid DNA (pDNA), a vital building block for mRNA-based vaccines, viral vector-based cell, or gene therapies that require pDNA as a starting material. .

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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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Drug Discovery World

OCTOBER 17, 2022

One critical bottleneck in Cell and Gene Therapy (CGT) manufacturing is the production of high-quality plasmid DNA (pDNA), a vital building block for mRNA-based vaccines, viral vector-based cell, or gene therapies that require pDNA as a starting material. .

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