Mission Bio, a pioneer in high-throughput single-cell DNA and multi-omics analysis, has completed its first tech transfer of a clinical trial-ready cell and gene therapy (CGT) assay from its Tapestri Platform to Avance Biosciences Inc., a CRO based in Houston, Texas, to assess transduction efficiency for an autologous cell therapy currently under development.
 
This collaboration marks the first time the Tapestri Platform will undergo qualification and validation within a good manufacturing practice (GMP) setting, paving the way for its routine use in clinical trials on CGT materials to support the next wave of life-saving CGT treatments.

The Tapestri Platform will allow Avance to analyze cells that have been transduced with lentivirus, identifying transgene integration within individual cells without the need to grow clonal populations. Tapestri analysis not only reduces the timeframe of conventional workflows, but also enables large sample sizes (at the scale of thousands of individual cells) to be rapidly assessed — yielding highly accurate and precise measurements of transduction efficiency.
 
Assessing the safety and efficacy of CGT products is more challenging than conventional therapies because of the many potential variations between genetically altered cells. Cells modified with a viral vector will differ in transduction efficiency and the number of copies incorporated into the genome. Cells modified with tools like CRISPR can vary in on- and off-target edits, zygosity, or aberrant translocations. Before Tapestri, workflows required synchronizing data from multiple genotypic and phenotypic assays over several weeks, an approach with reduced specificity and limited ability to characterize cells with simultaneous edits.
 
The qualification and adoption of Tapestri assays – already used in pre-clinical settings to assess critical quality attributes of cell and gene therapy candidates – in a GMP environment validates their utility at the clinical and chemistry, manufacturing, and control (CMC) phases. With GMP compliance, Tapestri can be used for batch release assays to assess products prior to administration to patients.

Comments from Leadership

“As the demand for cell and gene therapies continues to increase, the need to understand which genomic modifications are taking place in which specific cells is paramount for ensuring clinical safety and efficacy,” said Yan Zhang, Ph.D., CEO of Mission Bio. “To our knowledge, this is the first high-throughput single-cell assay in use for clinical-grade cell therapy manufacturing. We’re excited to see how a partner with Avance’s experience and reputation for high-quality GMP compliant assay validation and its client harness Tapestri’s capabilities.”
 
“We are excited to add another state-of-art technology to our biological assay toolkit to support our ever-growing base of clients for their gene and cell therapy development initiatives,” said Xuening (James) Huang, Ph.D., co-founder and CEO of Avance. “Tapestri will allow us to assess transduction efficiency at single-cell resolution, skipping laborious steps that delay these therapies from patients who are in dire need.”