Eurofins Regulatory & Consultancy Services Italy’s Post

🧬📢 New FDA Draft Guidance: A Must-Read for Gene Therapy Developers!   In November 2024, the FDA issued a draft guidance document that addresses frequently asked questions about developing cellular and gene therapy products. This guidance is designed to assist the industry in overcoming common challenges and includes key areas such as: 🧬 FDA Interactions: IND submissions, meeting types, amendments, and expedited programs 🧬 CMC Considerations: Product characterisation, critical quality attributes, analytical methods, process validation, manufacturing changes, and stability 🧬 Non-Clinical Studies: Animal model selection, proof-of-concept studies, toxicology, and pharmacology 🧬 Human Trials: Trial design, endpoint selection, and safety data   Read full document: https://lnkd.in/es726_NG   Need support? 🤝 With our extensive expertise in CMC/quality both regulatory and testing support, as well as our pre-clinical knowledge, we are uniquely positioned to help you navigate these new guidelines effectively and elevate your development of cellular and gene therapy products.   What challenges have you faced in developing gene therapy products? Let us know in the comments! 👇   Innovate with confidence and contact us today: Eurofins.Biolab@bpt.eurofinseu.com #CellTherapy #FDA #CGT #DrugDevelopment #GeneTherapy #Therapeutics

📢 NEWS | FDA Draft Guidance on FAQs for Cellular and Gene Therapy Products ▶ On November 18, the FDA released the draft guidance: "Frequently Asked Questions: Developing Potential Cellular and Gene Therapy Products." https://bit.ly/3ARrseR ↪ This document addresses common #questions and #challenges faced by the industry during the #development of cellular and gene therapy (#CGT) products. Its goal is to facilitate the creation of #safe, #effective, and high-quality #therapies. ↪The #FAQs are based on inquiries from sponsors, feedback following #FDA public presentations, and input from stakeholders via #CBER. They cover critical areas such as: 🔹 Regulatory review 🔹 Chemistry, Manufacturing, and Controls (CMC) 🔹 Pharmacology/Toxicology 🔹 Clinical considerations 🔹 Clinical pharmacology ↪The guidance is open for public comment and may be updated in the future to include additional FAQs. 💡 Need support navigating this guidance or other regulatory requirements? Contact us at info@asphalion.com to learn how we can help. #Pharma #Drugs #Medicines #FDA #CGT #CellularTherapies #GeneTherapies #RA #PharmaceuticalIndustry #FDA #RegulatoryAffairs #WeAreAsphalion #WeCare #KnowledgeFromExperience https://bit.ly/4hWWSkF

📢 NEWS - FDA Draft Guidance on FAQs for Cellular and Gene Therapy Products ▶ On November 18, the FDA released the draft guidance: "Frequently Asked Questions: Developing Potential Cellular and Gene Therapy Products." https://bit.ly/3ARrseR ↪ This document addresses common #questions and #challenges faced by the industry during the #development of cellular and gene therapy (#CGT) products. Its goal is to facilitate the creation of #safe, #effective, and high-quality #therapies. ↪The #FAQs are based on inquiries from sponsors, feedback following #FDA public presentations, and input from stakeholders via #CBER. They cover critical areas such as: 🔹 Regulatory review 🔹 Chemistry, Manufacturing, and Controls (CMC) 🔹 Pharmacology/Toxicology 🔹 Clinical considerations 🔹 Clinical pharmacology ↪The guidance is open for public comment and may be updated in the future to include additional FAQs. 💡 Need support navigating this guidance or other regulatory requirements? Contact us at info@asphalion.com to learn how we can help. #Pharma #Drugs #Medicines #FDA #CGT #CellularTherapies #GeneTherapies #RA #PharmaceuticalIndustry #FDA #RegulatoryAffairs #WeAreAsphalion

📢 NEWS - FDA Draft Guidance on FAQs for Cellular and Gene Therapy Products ▶ On November 18, the FDA released the draft guidance: "Frequently Asked Questions: Developing Potential Cellular and Gene Therapy Products." https://bit.ly/3ARrseR ↪ This document addresses common #questions and #challenges faced by the industry during the #development of cellular and gene therapy (#CGT) products. Its goal is to facilitate the creation of #safe, #effective, and high-quality #therapies. ↪The #FAQs are based on inquiries from sponsors, feedback following #FDA public presentations, and input from stakeholders via #CBER. They cover critical areas such as: 🔹 Regulatory review 🔹 Chemistry, Manufacturing, and Controls (CMC) 🔹 Pharmacology/Toxicology 🔹 Clinical considerations 🔹 Clinical pharmacology ↪The guidance is open for public comment and may be updated in the future to include additional FAQs. 💡 Need support navigating this guidance or other regulatory requirements? Contact us at info@asphalion.com to learn how we can help. #Pharma #Drugs #Medicines #FDA #CGT #CellularTherapies #GeneTherapies #RA #PharmaceuticalIndustry #FDA #RegulatoryAffairs #WeAreAsphalion

Pfizer announced on Wednesday morning that its gene therapy for hemophilia A met its primary objective in a key clinical trial, and the company plans to bring the data to health regulators for approval in the upcoming months. The gene therapy, known as giroctocogene fitelparvovec, was non-inferior to routine factor VIII replacement therapy in reducing bleeding rate, meeting the primary endpoint of the trial. The gene therapy showed it was better than factor VIII replacement therapy at reducing bleeding in a superiority analysis, according to Pfizer, and significantly reduced bleeding rates in patients compared to before their gene therapy infusions.... #genetherapy #cellandgenetherapy #advancedtherapies #regenerativemedicine #hemophiliaA #clinicaldata #primaryendpointsmet #submittingforapproval #reducedbleeding #raredisease #lifesciences #lifescience #biotech #biotechnology #pharma #pharmaceuticals #FDA #PhaseIII #clinicaltrials

Watch our webinar, 'Assessing extractables & leachables' role in ensuring the safety of cell and gene therapy product manufacture and long-term storage,' led by industry expert Nick Morley. https://ow.ly/oO0c50QOTqU Explore critical testing nuances and regulatory changes in this pre-recorded webinar. Watch to understand the challenges in leachable risk assessment, learn how to design and execute Extractable & Leachable studies for advanced therapy medicinal products (ATMPs) in disease treatment, the impact of solvents on E&L profiles, and navigate the evolving regulatory landscape for long-term storage of cell and gene therapy products. Access the webinar today! #CellAndGeneTherapy #EandLTesting #Webinar #Pharmaceuticals #MedicalDeviceTesting #LifeSciences #PharmaceuticalTesting

𝗧𝗵𝗲 𝗰𝗲𝗹𝗹 & 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆 𝗯𝗶𝗼𝗮𝗻𝗮𝗹𝘆𝘁𝗶𝗰𝗮𝗹 𝘁𝗲𝘀𝘁𝗶𝗻𝗴 𝘀𝗲𝗿𝘃𝗶𝗰𝗲𝘀 𝗺𝗮𝗿𝗸𝗲𝘁 𝘀𝗶𝘇𝗲 𝗶𝘀 𝗲𝘅𝗽𝗲𝗰𝘁𝗲𝗱 𝘁𝗼 𝗿𝗲𝗮𝗰𝗵 𝗨𝗦𝗗 𝟵𝟱𝟬.𝟬𝟲 𝗺𝗶𝗹𝗹𝗶𝗼𝗻 𝗯𝘆 𝟮𝟬𝟯𝟮,𝗲𝘅𝗵𝗶𝗯𝗶𝘁𝗶𝗻𝗴 𝗮 𝗖𝗔𝗚𝗥 𝗼𝗳 𝟳.𝟯% 𝗱𝘂𝗿𝗶𝗻𝗴 𝟮𝟬𝟮𝟰–𝟮𝟬𝟯𝟮. 𝙈𝙤𝙧𝙚 𝙍𝙚𝙥𝙤𝙧𝙩 𝘿𝙚𝙩𝙖𝙞𝙡𝙨: https://lnkd.in/dwydCn-c The growing number of studies revealing the ability of cell and gene therapies to treat multiple diseases is driving investments in research activities in the healthcare industry. The government funding to increase its capacity to test medication and healthcare equipment in the marketplace. The rising pharmaceutical companies' initiatives to increase investments in the design and production of cell and gene therapies are driving the demand for performance-evaluating tools and services, which is positively influencing the global market. #cellandgenetherapy #bioanalyticaltesting #genetherapymarket #celltherapy #biopharma #biotechnology #biotechinnovation

A recent paper, "GENE THERAPY REVOLUTION: STRATEGIC IMPLICATIONS FOR BIOPHARMACEUTICAL VALUATION," delves into the transformative potential of gene therapy and its impact on the biopharmaceutical industry. This technology holds immense promise for treating a wide range of diseases, from rare genetic disorders to chronic illnesses. As gene therapy trials show success and reach the market, it's crucial for biopharma professionals to understand the strategic implications for valuation. Key points to consider: Shifting paradigms: Gene therapy could disrupt traditional treatment models, impacting how we value biopharmaceutical companies. Long-term vs. short-term focus: The potential for one-time curative treatments may require adjusting valuation methods to account for long-term benefits. Evolving regulatory landscape: Regulatory pathways for gene therapy are still developing. Understanding these complexities is essential for accurate valuation. Additionally, the paper explores the challenge of balancing the high cost of gene therapy development with its long-term benefits for patients and healthcare systems. #genetherapy #biopharma #valuation #healthcare #futureofmedicin

Excited to go first-in-human with your 𝐧𝐞𝐰 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲?🧬 Here's how to out-pace competitors & why 𝐟𝐨𝐫𝐦𝐮𝐥𝐚𝐭𝐢𝐨𝐧 matters: 𝐎𝐊, 𝐛𝐚𝐬𝐢𝐜𝐬 𝐟𝐢𝐫𝐬𝐭: 1) Before going in humans, you have to 𝐬𝐡𝐨𝐰 𝐭𝐨 𝐫𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐬 that your drug product is 𝐬𝐭𝐚𝐛𝐥𝐞 over the course of your first-in-human study. 2) Your have 𝐲𝐨𝐮𝐫 𝐟𝐢𝐫𝐬𝐭 𝐆𝐌𝐏-𝐛𝐚𝐭𝐜𝐡, ready for clinics: you set your product formulation in stone and are 𝐡𝐚𝐩𝐩𝐲 to see first stability data.🥳 3) At this point, you already lost any speed advantage. 😞 𝐖𝐡𝐲? 💡The "happy-stability-data" 𝐜𝐞𝐥𝐞𝐛𝐫𝐚𝐭𝐢𝐨𝐧 𝐩𝐚𝐫𝐭𝐲 should have happened 𝐥𝐚𝐬𝐭 𝐲𝐞𝐚𝐫 with data from non-clinical & engineering lots. Because your 𝐟𝐨𝐫𝐦𝐮𝐥𝐚𝐭𝐢𝐨𝐧 𝐬𝐜𝐢𝐞𝐧𝐭𝐢𝐬𝐭 should have made sure you are all set. He/She: 1️⃣ pushed to 𝐩𝐢𝐜𝐤 a formulation early and 𝐬𝐭𝐢𝐜𝐤 to it over the course of development. 2️⃣chose a drug product 𝐜𝐨𝐧𝐜𝐞𝐧𝐭𝐫𝐚𝐭𝐢𝐨𝐧 𝐥𝐞𝐯𝐞𝐥 that covers all possible clinical in-use scenarios. 3️⃣made sure to choose a 𝐩𝐫𝐢𝐦𝐚𝐫𝐲 𝐩𝐚𝐜𝐤𝐢𝐧𝐠 𝐦𝐚𝐭𝐞𝐫𝐢𝐚𝐥 (𝐯𝐢𝐚𝐥𝐬) that are in line with those where your GMP-batch is filled into. Want clever tips for your gene therapy development? FDA spills all the beans: 📖Have a look at FDA's new draft guidance: "𝐅𝐫𝐞𝐪𝐮𝐞𝐧𝐭𝐥𝐲 𝐀𝐬𝐤𝐞𝐝 𝐐𝐮𝐞𝐬𝐭𝐢𝐨𝐧𝐬 — 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐢𝐧𝐠 𝐏𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥 𝐂𝐞𝐥𝐥𝐮𝐥𝐚𝐫 𝐚𝐧𝐝 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐏𝐫𝐨𝐝𝐮𝐜𝐭𝐬." 🔗Link here: https://lnkd.in/d-dVKRXm It covers everything from 𝐈𝐍𝐃 𝐬𝐮𝐛𝐦𝐢𝐬𝐬𝐢𝐨𝐧𝐬 and 𝐩𝐫𝐨𝐝𝐮𝐜𝐭 𝐜𝐡𝐚𝐫𝐚𝐜𝐭𝐞𝐫𝐢𝐳𝐚𝐭𝐢𝐨𝐧 to 𝐜𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐭𝐫𝐢𝐚𝐥 𝐝𝐞𝐬𝐢𝐠𝐧 and 𝐥𝐨𝐧𝐠-𝐭𝐞𝐫𝐦 𝐬𝐚𝐟𝐞𝐭𝐲 𝐦𝐨𝐧𝐢𝐭𝐨𝐫𝐢𝐧𝐠. Key highlights include: 🔹 Guidance on interacting with the FDA, including IND submissions and meeting types. 🔹 Insights into product development considerations like critical quality attributes and manufacturing changes. 🔹 Recommendations for nonclinical and clinical trial designs. The document is open for public comment now. #AAV #genetherapy #formulation

🚀 Transform the Future of Viral Vectors in Cell & Gene Therapy Development! 🚀 Join us for a game-changing webinar on December 10th at 11 AM EST to dive deep into how platform processes revolutionize cell and gene therapy (CGT) development and manufacturing. Learn how these strategies can help accelerate timelines, cut costs, and enhance scalability, driving quicker access to life-saving therapies. 🧬💡 Key Takeaways:- 1. Advantages & challenges of using platform approaches in CGT 2. Insights into the FDA’s new Platform Technology Designation Program 3. Regulatory implications of adopting a platform strategy 4. Explore Thermo Fisher Scientific’s Rapid Development Framework—a powerful alternative to traditional processes for viral vectors and cell therapies Meet the Industry Experts in a Free Live Webinar: 🎤 Pouria Motevalian, Ph.D. – Director, Process & Analytical Development, Thermo Fisher Scientific 🎤 Jesse Bishop – Senior Manager, Regulatory Affairs, Thermo Fisher Scientific 🎤 Samira Shore – Director, Technical Program Design, Thermo Fisher Scientific This webinar is essential for pharmaceutical and biotech professionals looking to optimize their CGT processes and stay ahead of the curve. 🗓 Register here for a Free live Webinar: 👇 https://lnkd.in/d4JBZRyy Secure Your Spot – Don’t miss out on expert insights and strategies to supercharge your CGT development! #CGT #GeneTherapy #Pharmaceuticals #Biotechnology #ThermoFisher #Webinar #Innovation #RegulatoryAffairs #ViralVectors