A New Platform for Clinical Gene Editing of iPSCs to Make Clonal Cell Banks

An emergent area of immuno-oncology and T-cell therapy is to make an “off-the-shelf” (or allogeneic) supply of suitably engineered T cells or natural killer (NK) cells so they can readily be administered across a wide variety of patients.

This supply can now be developed from suitable donor-patient induced pluripotent stem cells (iPSCs), and the first iPSC-derived CAR-T therapies are already enjoying success in the clinic.

However, somewhat analogous to monoclonal antibodies 20 years ago, the editing and single-cell cloning of stem cells has, up until now, largely been a black art reserved for experts. There is also great variation between groups and labs. Stem cells are inherently very sensitive to fluorescence-activated cell sorting (FACS) and culture conditions and their viability is typically poor when cloned. Current cloning approaches include colony picking and two rounds of cloning, which is very time-consuming.

Solentim now presents a platform combination of instrumentation (the VIPS) and soluble matrix (MatriClone, based on laminin) for the high-efficiency, high-viability single-cell cloning of iPSCs to form the Master Cell Bank. This includes an industry-leading clonality documentation package to support future investigational new drugs (INDs).

Most current instrumentation for stem cell isolation and cloning is only suitable for use in a research environment. Solentim realizes the need for cell therapy customers to implement systems in research now which can then be replicated and used in their clinical manufacturing. Therefore, Solentim aims to ensure all equipment is supplied in line with global cGMP requirements. That said, their equipment is fully validated according to cGMP and specific GAMP 5 principles and regulatory requirements for computerized systems, such as 21 CFR (Code of Federal Regulations) part 11 and EudraLex Volume 4, Annex 11. This is deemed as essential for customers planning to adopt this approach in their GMP manufacturing environment.

Illustrative data and images will be presented to demonstrate data quality assurance and integrity, process improvements, time and cost savings. Operational cGMP considerations will also be presented.

This platform will allow for groups to more easily perform clinical gene editing and cloning of iPSCs across a broader range of cell therapies, including neurodegenerative diseases, diabetes, heart failure and hair loss. It facilitates a more reproducible process for making clonal iPSCs, the ability to standardize the process, help establish and meet the future regulatory expectations for clonality and make the number of gene editing projects far more scalable.

Register for this webinar to learn about a novel platform for easier clinical gene editing and iPSC cloning.

Speakers

Dr. Ian Taylor, Chief Business Officer, Solentim Limited

Dr. Taylor has a Ph.D. in Biochemistry. He has more than 25 years of experience working in the conceptualization and commercialization of novel high-value life science instrumentation.

Prior to joining Solentim when it was founded ten years ago, Dr. Taylor was the Commercial Director at Genetix PLC, where he was responsible for the development and sales of the ClonePix FL for selection and isolation of high-value monoclonal-antibody-producing clones. Prior to that, Dr. Taylor was part of the senior leadership team for PerkinElmer Life Sciences.

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Simon Hoffman, GMP Consultant

Simon is a Principal GMP Consultant with over 25 years’ experience in the pharmaceutical, medical device and healthcare industries. Having started his career in world-class companies such as Novartis and AstraZeneca, Simon has an extensive track record of designing and setting up large-scale manufacturing sites and laboratories and launching healthcare products globally, all in line with world-class standards, including FDA, MHRA and ISO standards. Simon’s most recent achievement was being a key adviser and hands-on key team member for the launch and scale up of a COVID-19 coronavirus vaccine in record time.

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