Cell & Gene Therapy Insights

Big pharma case study: Alpha testing of a generic anti-#AAVkit and comparison to in-house Methods #Genedelivery via #AAVs has emerged as a pivotal technology in the clinical treatment of human diseases. However, the high prevalence of #antiAAVantibodies, which can diminish the effectiveness of the therapy, necessitates assessing levels of pre-existing #immunogenicity for individual patients. Next month, Gyros Protein Technologies’ Kira Hedström (Application Scientist) and Bayer’s Eva- Maria Thüring (Postdoc, Bioanalytical Department) will present a #casestudy comparing a #genericassay designed to detect anti-AAV antibodies with results obtained from in-house validated #ELISAplateassays Register below:

#Article now available! From the lab to the bench: scaling up regulatory #Tcelltherapy Regulatory #Tcells offer advantages for #immunemodulation in #celltherapies due to their multiple mechanisms of action, including regulation of antigen-presenting cells and #Tcelleffector function. This article addresses the key challenges in the evolving field of #Tregbasedtherapy, emphasizing safety, accessibility, and manufacturing efficiency, and explores strategies such as the implementation of automation and closed systems to enhance operational consistency and minimize contamination risks. Read more below: https://buff.ly/3OYYuNs

#eBook now available! Highlights from the Cell and Gene Therapy Summits: what’s next for #advancedtherapies? From scaling up manufacturing to streamlining regulation, the industry is gearing up for the next generation of cell and #genetherapies. Based on the popular Cell & #GeneTherapy Summit events, this interactive eBook summarizes key insights from academia and #biotech, investors, regulators, solutions providers, and patient advocates. Using succinct summaries, audio, and #infographics, the eBook outlines the current landscape and what’s next for the field in terms of scientific advances, regulation, manufacturing, and commercial viability Access below:

#Webinar now available on demand! Accelerating #AAVproduction: optimizing compound identification and process parameters The high cost of #genetherapies poses significant challenges in both bringing these life-saving treatments to the market and ensuring patient access. A major factor driving these costs is insufficient production yields, highlighting the need for novel technologies to scale up #AAVmanufacturing. Virica Biotech’s Keara Sutherland (Scientist) and Culture Biosciences’ Jean-Marc Guedon (Director, Application Technologies) present novel workflows that accelerate the identification of compounds that enhance AAV production and discuss how a cloud-connected #bioreactor platform facilitates the transition from lab-scale to large-scale #genetherapymanufacturing Watch at your convenience below:

Accelerating #AAVprocessdevelopment with a PAT-driven #TFFsystem #Ultrafiltration/#Diafiltration (UF/DF) represents a crucial step in bioprocessing, but traditional methods can pose various challenges related to variability and lack of real-time monitoring, which necessitates the adoption of novel analytical technologies. The following recently published content describes how a process analytical technology (PAT)-driven system provides UF/DF process monitoring and control through real-time concentration measurements and set points, particularly focusing on the #AAV2serotype. View below:

#Webinar now available on demand! Process in the product: a new analytical tool for #AAVproduction optimization and monitoring #Chromatographicanalysis of complex #AAVvectors presents challenges due to the presence of various impurities, including proteins, DNA, and other matrix species that may coelute with the target molecule. In cases where one-dimensional #chromatography does not allow satisfactory separation, more efficient analytical tools are required to address these issues. Sartorius BIA Separations’ Blaž Bakalar (Product Manager PATfix® Platform) and Ivana Petrović Koshmak (Head of Upstream Process Development) explore the determination of the percentage of #fullcapsids in complex samples, including lysed harvests and spent media, with rapid analysis. Watch at your convenience below:

#Webinar now available on demand! When does outsourcing #autologous collection center qualification and the #celltherapy supply chain make sense? Some #CARTtherapy sponsors have developed in-house teams to manage, improve, and maintain the infrastructure needed for these complex therapies, while others outsource these responsibilities. It is crucial to have a basic framework to evaluate which method is more suitable for a particular product. Cryoport Systems’s Amy Hines (Vice President, Apheresis Strategy), NMDP BioTherapies’ Jeni Newman (Senior Director, Operations), and Orchard Therapeutics’ Marcel van Houten (Director of Global Supply Chain) review the successes and challenges of both in-house and outsourced models based on their extensive experiences and compare various approaches to building a collection center network. Watch at your convenience below:

As the #genetherapy field advances, speed-to-market is becoming more and more essential. The necessary reduction in development timelines can be realized by leveraging platform-based tools for #AAVmanufacturing and choosing optimal affinity resins. Recently published content covering the advancements in #AAVvectorpurification and insights on future developments in the downstream process of this gene therapy #vector includes: An article and webinar exploring the use of a novel #AAVaffinity capture resin as part of an off-the-shelf AAV production system for various serotypes, and a case study using novel platforms to capture AAV vectors from crude material with high purity and high recovery for a broad range of serotypes. Article: https://buff.ly/4gzEMUd Webinar : https://buff.ly/3ZH6API These are just a few of the resources available on our Purification Hub. The hub explores the key role that viral vectors and mRNA play in cell and gene therapy, the current challenges facing the field, and potential solutions for successful manufacture. Once you’ve read your first piece of content, all the rest is available to you open access. https://buff.ly/3T12nDj

Generation of novel #AAVserotypes with enhanced infectivity, specificity, and lower toxicity via #AAVcapsidengineering platform Capsid engineering can enable successful development of novel AAV serotypes with enhanced specificity, infectivity, and reduced toxicity. In this article, a #capsidengineering platform is described, along with study data on promising #AAV variants generated using this approach. Read below:

#Webinar now available on demand! How to use #AI and #machinelearning in #celltherapy process development The cell therapy field is progressively shifting towards the digitization and automation of the entire development process. Additionally, the rise of AI and machine learning poses new opportunities, especially for analyzing data sets during #celltherapymanufacturing. In this webinar, Charles River Laboratories’ Alex Sargent (Director of Process Development) discusses how to best utilize AI and ML for cell therapy process development, and introduces #DoE and a case study on optimizing perfusion in allogeneic #Tcellmanufacturing with these technologies. Watch at your convenience below: