QIAGEN Life Sciences’ Post

I had the privilege of joining this workshop virtually. There was collaborative discussion with FDA, industry, and academia around what could and should be used as non-proprietary “building blocks” in CGT manufacturing to help streamline costs and make products more accessible to patients. Potential building blocks discussed for AAV included cell banks, unit operations like bioreactor or purification processes, and standardized analytics and reference material. The intent is to have elements that can be transportable across different programs but are not as formal as FDA’s designated “platform technologies” which only apply to licensed products. In his closing remarks, Peter Marks indicated that AAV manufacturers need to realize that innovation is not in the columns and buffers used for manufacturing, but in the construct itself. This resonated with me since I see so much competition in the CDMO space over manufacturing platforms and proprietary reagents, when the real value of a manufacturing process depends on the construct and delivery approach of the gene therapy product. We should focus instead on leveraging the platform-product fit and sharing best practices and knowledge to ensure enough of the right products get manufactured and released to the patients who need them. I’m looking forward to this white paper coming out next year, as well as output from other precompetitive consortia like BioPhorum and FNIH. #genetherapy

What if there are modular components of CGT development that can be leveraged across developers to save time and resources? What do these "building blocks" look like, and how can they be disseminated within the field? Earlier this month, ARM convened key stakeholders from the CGT industry and held a Scientific Exchange meeting with FDA around this building block concept to streamline development and manufacturing, with case studies across three technology areas (AAV, LNP, and iPSCs). Check out the meeting preview here: https://lnkd.in/gvtxhvGq The full comprehensive white paper will be published early next year!

🎉 Exciting news alert! 🚀 Our amazing team has launched Biozen dSEC7 column, and I couldn't be more thrilled to share it with you all. This innovative product is set to revolutionize Biopharma industry, and I can't wait to see the impact it makes. #NewProduct #Innovation #Teamwork #biopharma #Biozendsec7

This week, we profile Dr. Sarah Hedtrich from the UBC Faculty of Pharmaceutical Sciences. Her lab combines her expertise in #pharmacology, #BiomedicalEngineering, and #DrugDelivery to develop the next generation of gene therapy. We discuss her recent publication in ACS Publications, the challenges of creating gene editing-based therapies, and the impact this could have on patients as Dr. Hedtrich sets her sights on clinical trials. 📖 : https://bit.ly/3GD0yGE

Highly recommended & convenient GoTo point…

Want to hear more about negotiating cell and gene therapy deals? We’ve got a great panel line up on 5 June so do sign up to hear more about their experiences when CGT licensing and collaboration agreements, investment rounds and more. I’m really looking forward to chairing this discussion! 😀🧬🏥 #bristowsllp, #onenucleus, #cellandgene, #intellectualproperty, #lifesciences

This draft guidance provides recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect. #fda #fdaapproval #fdaregulations #fdacompliance #regulatoryaffairs #regulatory #genetherapy #cellulartherapy

Only 3 weeks left until the 4th Gene Therapy Comparability Summit! With over 2,000 gene therapies in the clinic and the FDA opening a 'super office,' it's more important than ever to understand and address comparability for gene therapies to ensure regulatory approval. Our expert speaker faculty, including Sarepta, Novartis, and AskBio, will delve deep into addressing current challenges. Joining us in the audience are also the likes of Spark Therapeutics, Regeneron Pharmaceuticals, and Genethon. Pre-conference and conference day passes are running low, so don't miss out! Take the time to have discussions internally and secure your spot. Agenda: https://lnkd.in/eWEn8JZ7 Register: https://ter.li/i52rly #GeneTherapy #ComparabilitySummit #FDA #RegulatoryApproval