Are you meeting safety standards for residual host cell DNA?
Bio Pharma Dive
JULY 1, 2024
Ensure the safety of gene therapies with accurate HEK293 DNA measurement using Vericheck ddPCR kits.
Sartorius upscales gene therapy prowess with $2.6bn Polyplus acquisition
Pharmaceutical Technology
APRIL 3, 2023
The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.
Gene Therapy 
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Gene Therapy and Pharmacokinetics
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
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CDMO Bionova branches into plasmid DNA to serve cell and gene therapy developers
Fierce Pharma
JUNE 3, 2024
subsidiary Bionova Scientific is launching a new business line to provide services around plasmid DNA (pDNA), the companies announced Monday. As part of the pDNA pivot, Bionova is setting up a new facility in The Woodlands, Texas.
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Synplogen and Ginkgo Bioworks sign MoU for DNA manufacture
Pharmaceutical Technology
DECEMBER 7, 2023
Synplogen and Ginkgo Bioworks have signed an MOU to expedite DNA manufacturing and gene therapy platform services in Japan.
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New facility boosts UK’s cell and gene therapy manufacturing capacity
Pharmaceutical Technology
MARCH 10, 2023
On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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How can long-read nanopore sequencing support gene therapy delivery?
Drug Discovery World
MAY 8, 2024
Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The post How can long-read nanopore sequencing support gene therapy delivery? The event will take place on 13 May, 4PM BST. appeared first on Drug Discovery World (DDW).
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GenScript ProBio and CCS partner for cell and gene therapies development
Pharmaceutical Technology
JUNE 8, 2023
GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. The core objective of the collaboration is to make the therapies affordable and accessible to patients.
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?5 Things You Need to Know from Our Recent Gene Therapy Webinar
Worldwide Clinical Trials
JUNE 15, 2022
At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.
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Gene therapy specialist bluebird exits “untenable” European market
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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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease
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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How to advance AAV-based gene therapies
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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Astellas takes $170m charge as it drops DMD gene therapies
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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Guide to using AAV vectors in gene therapy
Drug Discovery World
FEBRUARY 8, 2024
In 2023, 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. Why are those important?
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Takeda grows in gene therapies again with $2bn Code Bio deal
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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Forge adds plasmid DNA manufacturing to integrate gene therapy production service
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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BioMarin Gene Therapy Halted by FDA Over Safety Concerns
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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Helixmith starts new trial of diabetic neuropathy gene therapy
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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New London facility exceeds global plasmid DNA demand
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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FDA slaps clinical hold on BioMarin’s PKU gene therapy
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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Guide to using AAV vectors in gene therapy
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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Ancient viruses may hold key to gene therapy treatments
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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Early phase support for plasmid DNA supply to secure future manufacturing
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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DDW event focusses on rAAVs and long-read nanopore sequencing
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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The challenges and trends of cell & gene therapies
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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FDA Approves Gene Therapy Adstiladrin for the Treatment of Bladder Cancer
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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Expert view: What’s next for cell and gene therapy?
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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bluebird seeks gene therapy trial restart after cancer scare
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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Q&A: Gene therapy opportunities from long-read sequencing
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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DNA Cloning Market: A Step Towards Future of Genetics Science
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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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics
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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Charles River’s expansion to Alderley Park set to bolster UK cell and gene therapy manufacturing capability
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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FDA lifts hold on uniQure gene therapy after cancer case review
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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FDA accepts GMP plasmid DMF: Andelyn now ‘on track’ to speed up gene therapy development
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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rAAV Genome sequencing uncovers transgene integrity and packaging impurities
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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Vertex signs licence deal with CRISPR Therapeutics for diabetes therapies
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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Uncovering packaging impurities with rAAV genome sequencing
Drug Discovery World
MAY 1, 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.
Packaging 52
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How rAAV genome sequencing uncovers transgene integrity
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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GenScript ProBio and RVAC partner for Covid-19 vaccine pDNA
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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Touchlight appoints Karen Fallen as CEO of Touchlight DNA Services
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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Global Viral Vector and Plasmid DNA Manufacturing Market (2020 to 2030) – Growth, Trends, and Forecasts – ResearchAndMarkets.com
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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Gene Editing Innovations with Programmable Gene Insertion (PGI) Technology ft. John Finn, PhD, CSO, Tome Biosciences – Xtalks Life Science Podcast Ep. 160
XTalks
MAY 22, 2024
In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.
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Webinar: How to resolve barriers to CGT discovery
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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Bluebird suspends gene therapy trials; Cortexyme tripped up by FDA hold; Guardant Health launches surveillance blood test; Enhertu scores approval
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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