Cell & Gene Therapy Insights

#Webinar now available on demand! Developing off-the-shelf #CARNKcells targeting #CD70: updates from preclinical and early clinical studies There are six FDA-approved CAR-T cell products, but their reliance on autologous #Tcells limits accessibility and scalability. To address this, the field is shifting towards #allogeneic platforms, but traditional use of T cells poses risks of graft-versus-host disease (GvHD) due to human leukocyte antigen recognition. MD Anderson Cancer Center’s Sunil Acharya (Principal Research Scientist) discuss the potential of off-the-shelf CAR natural killer (NK) #celltherapy, illustrating its efficacy, safety, and promise in making cancer treatment more accessible and scalable. Watch at your convenience below:

From the lab to the bench, scaling up regulatory #Tcelltherapy #RegulatoryTcells (#Tregs) are crucial for maintaining #immunebalance and preventing overactive #immuneresponses. While their therapeutic use is a rapidly expanding field of research, producing Tregs presents significant challenges, including contamination risks and human error. Next week, Thermo Fisher Scientific’s Hany Meås and the University of British Columbia’s Megan K. Levings will discuss the growing potential of Treg #celltherapy for treating #autoimmuneconditions, and the considerations for producing these therapeutics in a closed, automated environment. Register for this #webinar below:

Addressing the challenge of screening #DNA-#LNPformulations for targeting the brain Developing hundreds of #lipidnanoparticle (#LNP) formulations for #invitroscreening is challenging, especially when using barcoding strategies to increase throughput. Additionally, transitioning the #LNPproduction process to the development stage is difficult due to equipment changes. Next week, join Karolinska Institutet’s Razieh Karamzadeh (Post-doc researcher, Division of Molecular Neurobiology) and Unchained Labs’ Ben Knappett (Market Manager – LNP Solutions) as they present data from experiments using novel systems for the production of DNA-LNPs for in vitro screening, then scaling-up selected LNPs for #invivostudies Register for this #webinar below:

#Article now available! #iPSC-based therapy dilemmas explored: overcoming hurdles for future success There are many dilemmas to address for sustained progress and commercial viability in the iPSC-based therapy industry In this article, industry experts delve into the critical aspects of reshaping the future landscape of #iPSCbasedtherapies, exploring key areas such as material access and readiness, operational intricacies, technological innovations, and standardization. Read at your convenience below:

#Article now available! Evaluation and performance of an #AAVaffinityresin: a #CDMO case study There are now six #AAV-based #genetherapy products on the market, with many more in the R&D pipeline, which has led to an increased demand for the development of efficient and robust downstream purification processes to ensure the production of high-quality AAV vectors. This article introduces POROS™ CaptureSelect™ AAVX affinity resins for the scalable #downstreampurification of a range of #AAVserotypes for #genetherapy applications. Read this article at your convenience below:

Enhancing CAR-T therapy development: harnessing cell selection flexibility Advancements in #CARTcelltherapy have driven the need for innovative, closed, automated, and space-efficient manufacturing platforms. Considering each therapeutic product requires a unique manufacturing process, flexibility in process design and automation is essential to accommodate these varying needs. Next month, Lonza’s Tamara Laskowski (Senior Director, Clinical Development) and Nuala Trainor (Director, R&D, Personalized Medicine) will introduce a novel automated manufacturing platform that can be adapted to different #CARTmanufacturing processes by positively or negatively selecting #Tcells directly from apheresis, and discuss the advantages associated with this approach. Register for this #webinar below:

#Article now available! It’s a match: #celllineengineering for #AAVmanufacturing expands the options for therapeutic programs This article will address how advanced engineered cell lines can empower researchers and developers to realize the full potential of #genetherapy. New methods to harness cell line engineering to improve #AAV through quality by design will be explored, alongside new performance data acquired with ELEVECTA™ #celllines. Read at your convenience below:

Key considerations for shortening your #CARTmanufacturing process Autologous CAR-T #celltherapy has demonstrated clinical efficacy in treating patients with B-cell malignancies. Given the critical condition of these patients, shortening the manufacturing time of CAR-T cells is crucial to improve therapeutic outcomes. Later this month, join Nadine Mockel-Tenbrinck (Manager for Process Engineering, CMC Research and NCD, Miltenyi Biotec) as she discusses how to establish a robust and automated 1-week manufacturing process for viable CAR-T cell products and presents #invitro and #invivo efficacy studies Register for this upcoming #webinar below:

#Article now available! Combining best practices and analytical reference materials to tackle challenges in #AAVmanufacturing The rapid increase in the gene therapy industry creates a high demand for #plasmids and other starting materials. However, manufacturing challenges and compressed development timelines make scaling production difficult from early development stages. Despite these challenges, #genetherapy products must still comply with existing regulations. This article discusses the ongoing efforts by organizations to create documentary standards and reference materials, facilitating consistent and predictable manufacturing processes for #AAVs. Read at your convenience below:

#Article now available! Considerations to optimize and act upon long-term follow-up studies for cell and gene therapies While both cell and #genetherapies (CGTs) offer long-term benefits for patients, safety and efficacy must be thoroughly evaluated before widespread clinical use. As a result, #CGTs almost always require long-term follow-up (LTFU) studies, starting in Phase I clinical trials. In this article, key regulatory, operational, medical, and scientific insights when considering #LTFU studies in the CGT space are discussed. Read at your convenience below: