World Pharmaceutical Frontiers’ Post

Mark your calendar for an insightful panel discussion with Dr. Rachel Legmann, Sr. Director of Technology, Gene Therapy at Repligen, and a lineup of industry experts. The discussion, titled “Phase-Appropriate Manufacturing: Trial and Error… or Trial and Triumph?”, will take place on September 24th at 4:30 PM during BPI Boston. Don’t miss this opportunity to hear from the experts about the key challenges facing manufacturing in the emerging cell and gene therapy industry Topics: - Navigating the complex grey area of manufacturing for Phase 1 and 2 trials - Keeping efficiency while complying with limited guidance - Need conversation between regulators and developers - Lessons learnt and case studies from companies moving between clinical phases - Therapeutic Developer/CDMO dynamics in the phase 1 and beyond - How to best leverage CDMO expertise and capital efficiency Join the discussion! https://lnkd.in/gt2mFKGN #BPIconf #bioprocessing #genetherapy #processdevelopment #repligen #monoclonalantibodies #vaccines

🧬 Investigating genome integrity of viral vector preparations just got easier. Learn how QIAcuity brings precision to your analysis of AAV genome integrity and beyond. Get the details in our new application note, entitled "Analysis of DNA integrity and stability using digital PCR," which you can download in our dPCR gateway to cell and gene therapy. Find it here https://lnkd.in/ebPfPKA5

Are currently facing regulatory challenges in your cell or gene therapies❓   Now's your chance to achieve seamless approvals in your first regulatory submissions with support from the 𝗖𝗲𝗹𝗹 & 𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗔𝗳𝗳𝗮𝗶𝗿𝘀 𝗦𝘂𝗺𝗺𝗶𝘁.   📚𝗥𝗲𝗮𝗱 𝘁𝗵𝗲 𝗙𝘂𝗹𝗹 𝗔𝗴𝗲𝗻𝗱𝗮 - https://ter.li/t7h8j0   Take this opportunity to learn from top regulatory experts at AstraZeneca, Bristol Myers Squibb, CytoImmune Therapeutics, and more as they delve into every aspect of cell and gene therapy regulation; from pre-IND to post-BLA, benchmarking best practices, and preparing for every critical milestone.   🔍𝗙𝗶𝗻𝗱 𝗼𝘂𝘁 𝗠𝗼𝗿𝗲 - https://ter.li/t7h8j0   We hope you'll join us in October, to understand guidance, streamline submissions, and obtain approval first-time.   #CellandGeneTherapyRegulatoryAffairsSummit 

"What are the current processes and procedures associated with cell and gene therapy development? Where are the bottlenecks, and why, and can we devise and prioritize solutions to overcome these bottlenecks? This roundtable evaluated these challenges from the regulatory pathway, clinical operations, and manufacturing perspectives." This DIA Global Forum article, co-authored by FDA's Peter Marks and Nicole Verdun and 11 other participants, summarizes numerous issues and proposed solutions discussed at the recent DIA/Harvard-MIT CRS Cell and Gene Therapy Executive Roundtable. #celltherapy #genetherapy #FDA #DIA #Harvard #MIT #regulatory #clinicaloperations #cellandgenetherapymanufacturing

Transforming Treatments: The New Era of Orphan Drugs is Here.🌟 Discover the groundbreaking advancements in orphan drug technology that are transforming rare disease treatment. From gene therapy to AI-driven innovations, the future is bright for patients and pharmaceutical companies alike. Explore how these developments are paving the way for hope and healing. https://lnkd.in/dgQdH-3K #OrphanDrugRevolution #RareDiseaseTreatment #InnovationsInOrphanDrugs #GeneTherapy #AIinOrphanDrugDevelopment

*** FDA’s List of Approved Gene Therapies *** Gene therapy is rapidly advancing, with several new FDA approvals paving the way for treatments that can potentially cure genetic disorders Recent and notable approvals: - Lenmeldy (atidarsagene autotemcel) by Orchard Therapeutics - U.S. Therapeutics for metachromatic leukodystrophy - Zynteglo (betibeglogene autotemcel) by bluebird bio for beta-thalassemia - Carvykti (ciltacabtagene autoleucel) by The Janssen Pharmaceutical Companies of Johnson & Johnson Biotech for multiple myeloma - Hemgenix (etranacogene dezaparvovec) by CSL Behring for hemophilia B - Roctavian (valoctocogene roxaparvovec) by BioMarin Pharmaceutical Inc. for hemophilia A #GeneTherapies #FDA #Healthcare #DataCollection

Our Head of CMC, Helen Sutton, attended the ‘Advances in Cell and Gene Therapies’ Symposium hosted by Melbourne Academic Centre for Health (MACH) on Monday, 13th of May. The event brought together experts to discuss the unique challenges and opportunities in cell and gene therapy. Key takeaways included the need for specialized skills, the potential for cost reduction through domestic capabilities, the importance of embedding quality early in development, and the necessity of increased funding to realize the full potential of these therapies. For expert advice, contact our Cell and Gene Therapy specialists at margaret@globalpharmasolutions.com. Follow us on LinkedIn to stay informed about the latest updates in regulatory affairs and industry insights. #cellandgenetherapy #celltherapy #genetherapy #biotechnology #pharmaceuticals #regulatoryaffairs #GlobalPharmaSolutions

The FDA just released a new 40-page draft guidance to streamline the path for developing and submitting cell and gene therapy (CGT) applications. 🎯 This Q&A-style document is packed with practical insights—from pre-IND meetings to long-term safety monitoring—and is designed to simplify the process for drugmakers navigating the complex CGT landscape. As part of the latest User Fee Reauthorization Act, this guidance underscores the FDA’s commitment to making cell and gene therapy development more efficient and accessible. In the agency’s own words: “This guidance is intended to support the development of CGT products by providing a repository of common questions posed to the Office of Therapeutic Products (OTP).” For anyone in the CGT space, this is a must-read! 🚀 #FDA #CellTherapy #GeneTherapy #Innovation

The #FDA has released the new draft guidance for industry, Considerations for the Use of Human- and Animal-Derived Materials in the Manufacture of Cellular and Gene Therapy and Tissue-Engineered Medical Products. This article by Dark Horse Consulting Group Inc.'s Blake Bergam and Sara Mills provides a summary — and shares what is shocking about and missing from the guidance and what to do about it. #cellandgenetherapy #cgt #draftguidance #regulatory https://lnkd.in/e5tehxHx

This #eBook, "Next-Generation Gene Therapies and AAV Products" addresses the production of next-generation adenoassociated virus (#AAV)–based products. Specifically, the Swiss #biopharmaceutical giant, Roche, hopes to break through the #manufacturing limitations that have limited the reach of such lifesaving treatments. Roche wants to create a range of #genetherapies that can treat large patient populations at affordable prices. Read the full eBook here: https://lnkd.in/gUuie-sC