Gene Engineering Services

Genome engineering is a mid-process step in cell therapy manufacturing. It takes place following isolation and activation of the desired immune cell population (e.g. T cells or NK cells). In gene editing, the cell’s genome is altered to generate a functional therapeutic cell population that will attack the patient’s disease. This may involve introduction of a disease-targeting chimeric antigen receptor (CAR) or TCR, inactivation of negative regulatory molecules, or alterations to enable the cells to avoid host immunity. Following genome engineering, the cells are expanded ex vivo, characterized, and infused into the patient.

Viral approaches (e.g. with lentivirus or AAV) involve transduction of the target cells, while non-viral transposons are electroporated into the target cells. Transposons have demonstrated a safe insertion profile without preference for any specific genomic sites. They maintain high efficiency gene transfer and stable expression, enabling rapid generation of transgenic mammalian cells with limited off-target effects.

TcBuster is the only commercially available transposon system, which means that its use is not limited by licensing restrictions. Bio-Techne’s genome engineering services with TcBuster address cell manufacturing process challenges by:

• Reducing the costs of genetic modification
• Providing cells engineered with GMP manufacturing controls in 4 months
• Improving the ability to integrate larger cargo sizes than practical with viral based methods
• Providing flexibility to utilize TcBuster with additional gene editing techniques in one operation

TcBuster shortens timelines and reduces the risk of delays. It is a valuable tool for both proof-of-concept studies and manufacturing scale process development.

Let Us Handle These Steps For You

• Consultation and preparation – speak with our gene editing experts who work with you to provide a solution that works for you.
• Isolation of your desired primary cells at small or large scale. We can obtain nearly any cell type  (e.g. CD34⁺ cells, T cells, B cells, MSCs) including specific donor characteristics (e.g. HLA type, sex, CMV status).
• Gene engineering/editing with any platform to generate high frequency gene knock-outs, knock-ins, or multiplex edits.
• Cell selection and expansion to maintain high functionality and viability (typically >95%).
• Validation of engineered cells – genetic (PCR), protein expression (Western blot or flow cytometry), and custom functional analysis.
• Cryopreservation with customized number of cells/vial and number of vials.

Challenges in iPSC Engineering

• Licensing complexities (e.g. consent and licensing from donors, licensing of gene integration methods). iPSC lines generated with non-integrating methods may not be approved for clinical use due to intellectual property restrictions.
• Extended timeline resulting from complexities and time requirements for optimization of iPSC generation

Our iPSC Processing Services for RUO, GMP, and Xeno-free Environments

• iPSC reprogramming, expansion, and master cell banking
• Gene knock-out
• Gene knock-in
• Multiplex gene editing (simultaneous knock-out/knock-in)
• Insertion or correction of single nucleotide polymorphisms (SNPs)
• HLA engineering to increase immunological compatibility with patient

Benefits of Our iPSC Cell Processing Services

• All services from gene editing to differentiation performed in-house
• Our cell culture experts have experience working under feeder, feeder-free, and xeno-free culture conditions
• We have a flexible platform to successfully edit cells from healthy or diseased donors
• We provide customizable validation assays and documentation
• Our dedicated project management team provides timely updates to inform progress of projects