Recommendations for Method Development and Validation of qPCR and dPCR Assays in Support of Cell and Gene Therapy Drug Development The emerging use of qPCR and dPCR in regulated bioanalysis and absence of regulatory guidance on assay validations for these platforms has resulted in discussions on lack of harmonization on assay design and appropriate acceptance criteria for these assays. Both qPCR and dPCR are extensively used to answer bioanalytical questions for novel modalities such as cell and gene therapies. Following cross-industry conversations on the lack of information and guidelines for these assays, an American Association of Pharmaceutical Scientists working group was formed to address these gaps by bringing together 37 industry experts from 24 organizations to discuss best practices to gain a better understanding in the industry and facilitate filings to health authorities. Herein, this team provides considerations on assay design, development, and validation testing for PCR assays that are used in cell and gene therapies including (1) biodistribution; (2) transgene expression; (3) viral shedding; (4) and persistence or cellular kinetics of cell therapies. https://lnkd.in/eEnj2D4f
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Mycoplasma contamination is a serious concern in cell and gene therapy product development. The FDA and Technical Guidelines for Pharmaceutical Research and Evaluation recommend mycoplasma control and testing for raw materials, intermediate samples, and final products. That's why Yeasen developed MycAway™ Mycoplasma qPCR Test Kit (probe method). This rapid, qualitative detection product based on NAT can detect potential mycoplasma contamination in raw materials, cell banks, viral seeds, virus or cell harvest fluids, and therapeutic cells. By using MycAway™, you can ensure the safety and quality of your cell and gene therapy products. Stay ahead of the game with Yeasen's innovative solution. https://lnkd.in/eZHR2aKT
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Check out the latest article we created for our partner BAP Pharma on our Pharmaceutical-Technology website - Medicines access: Unlocking the immense potential in cell and gene therapy Despite several ground-breaking developments, pharmaceutical and biotech companies are often reluctant or unable to invest in cell and gene therapy. Here, we discuss the key challenges to access and how the potential of these innovative treatments can be unlocked. - https://lnkd.in/djQmDiqb If you would like more information about our award winning content and account based marketing solution, please request a demo here: https://lnkd.in/e8wNB3cj #pharma #marketaccess #medicinesaccess #healthcaremarketing #digitalmarketing #contentmarketing #abm #accountbasedmarketing
Medicines access: Unlocking the immense potential in cell and gene therapy - Pharmaceutical Technology
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Another great case study from GlobalData Marketing Solutions and our partner BAP Pharma. Read more below. Book a demo of our latest Screener tools to find prospects and customers. Talk to me about your global B2B marketing content and lead generation needs. #AI-driven #marketingb2b #datadriveninsights
Check out the latest article we created for our partner BAP Pharma on our Pharmaceutical-Technology website - Medicines access: Unlocking the immense potential in cell and gene therapy Despite several ground-breaking developments, pharmaceutical and biotech companies are often reluctant or unable to invest in cell and gene therapy. Here, we discuss the key challenges to access and how the potential of these innovative treatments can be unlocked. - https://lnkd.in/djQmDiqb If you would like more information about our award winning content and account based marketing solution, please request a demo here: https://lnkd.in/e8wNB3cj #pharma #marketaccess #medicinesaccess #healthcaremarketing #digitalmarketing #contentmarketing #abm #accountbasedmarketing
Medicines access: Unlocking the immense potential in cell and gene therapy - Pharmaceutical Technology
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Thoughts on this? >> Only one Louisiana hospital to offer 'potentially life-saving' sickle cell gene therapies - NOLA.com >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #healthcare #biotech #competitivemarketing
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Metagenomi Announces Preclinical Data for Lead Hemophilia A Program Demonstrating Durable Factor VIII (FVIII) Activity Levels through Twelve Months Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, recently announced data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001. The twelve-month durability data from Metagenomi’s ongoing nonhuman primate (NHP) study in hemophilia A remains generally consistent with data previously released at 4.5 months. The NHPs remain healthy and exhibit normal weight gain. The treatment is generally well tolerated and the program is on track for IND filing in 2026. https://lnkd.in/g3XAnmgQ
Metagenomi Announces Preclinical Data for Lead Hemophilia A Program Demonstrating Durable Factor VIII (FVIII) Activity Levels through Twelve Months | Metagenomi, Inc
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🚀An insightful Harvard Business Review article that highlights how technological advancements are transforming the field of cell and gene therapies. In recent years the development of advanced therapeutic modalities (ATMs) like CRISPR-Cas9 and viral vectors has surged, enabling precise gene editing and cell reprogramming. These innovations are crucial to create effective treatments for previously untreatable diseases. 🤖 Automation and robotics are making the production of these therapies more scalable and cost-effective, while big data and AI are accelerating research, optimizing clinical trials, and personalizing treatments based on genetic information. This shift from a one-size-fits-all approach to personalized medicine is ultimately improving patient outcomes. 📈 The cell and gene therapy market is experiencing rapid growth, with an expected compound annual growth rate (CAGR) of around 25% over the next few years, and a market value forecast reaching $60 billion by 2027. This growth is primarily driven by technological advancements and increasing investment in R&D. 💡 These biotechnological advancements are not only enhancing the efficacy and accessibility of cell and gene therapies but also transforming the pharmaceutical industry’s approach to drug development and patient care. ⚠️ However, despite the promising advancements, there are still significant challenges. These include high costs of development and treatment, complex regulatory environments, and the need for specialized manufacturing facilities. Additionally, ensuring equitable access to these therapies remains a critical issue. 🌟In conclusion, while the field of ATMPs is rapidly advancing and holds immense promise for treating previously untreatable diseases, it is essential to address the existing challenges to fully realize its potential. Continued innovation, investments, and stakeholders’ collaboration across industries will be crucial in overcoming these hurdles and ensuring that these groundbreaking therapies are accessible to all who need them. #HealthcareInnovation #Biotechnology #MedicalResearch
A New Generation of Drug Therapies Requires New Business Strategies
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Thoughts on this? >> Researchers publish final results of key clinical trial for gene therapy for sickle cell disease - Medical Xpress >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #competitivemarketing #pharmaceutical #healthcare #pharma #biotech
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A big step in realizing our strategy towards on integrated offering for viral vector manufacturing!
Merck has signed a definitive agreement to acquire life science company Mirus Bio for US$ 600 million (around € 550 million). Mirus Bio is a specialist in the development and commercialization of transfection reagents. These reagents play a key role in the production of viral vectors for cell and gene therapies. The acquisition will advance our integrated offering for viral vector manufacturing and complements the existing portfolio for development and production of novel modalities – a key growth area for Merck Life Science. More: https://lnkd.in/eeF5HfRf [📷 from our Carlsbad, California manufacturing facility]
Definite-Agreement-Mirus-Bio | Merck
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Thoughts on this? >> Another Study Finds CRISPR Gene Therapy Fights Sickle Cell - Citizentribune >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #biotech #competitivemarketing #pharmaceutical #healthcare
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#News: MilliporeSigma , the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany, today announced the signing of a definitive agreement to acquire Mirus Bio for US$ 600 million. Mirus Bio, part of Gamma Biosciences , is a life science company based in Madison, Wisconsin, USA that specializes in the development and commercialization of transfection reagents, such as TransIT-VirusGEN®. Transfection reagents play a critical role in the production of viral vector-based gene therapies. “Viral vector-based cell and gene therapies have demonstrated their potential with more than 20 approvals over the last 10 years1 and will continue to advance, with a projected growth of 30 percent until 20282,” said Matthias Heinzel, Member of the Executive Board and CEO Life Science, Merck KGaA, Darmstadt, Germany. “We have been driving innovation in nucleic acid delivery for two decades,” said Dale Gordon, CEO of Mirus Bio. “MilliporeSigma’s broad portfolio, scale, and global reach, combined with our leading transfection reagents, will help take our business to even greater heights and allow us to serve more customers, and ultimately patients, worldwide.” Read more from Morningstar 👇🏽 https://lnkd.in/eZ9RPCNe
MilliporeSigma Signs Definitive Agreement to Acquire Mirus Bio, Furthering Ambition to Be a Leading Supplier for Viral Vector Manufacturing
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Innovative Team Lead and R&D Scientist | Expert in Gene Therapy, Biologics, Epigenetics, RNA | Collaborative and Creative Problem Solver
5moPunam Ghosh, PhD and Deepa Saxena