Mission Bio's single-cell transduction assay adopted for AVROBIO's gene therapy pipeline

 

New assay measures lentiviral vector transduction efficiency in days

AVROBIO to present data on the transduction assay at 4th Annual Gene Therapy Analytical Development Summit

SOUTH SAN FRANCISCO, Calif., Nov. 29, 2022 /PRNewswire/ -- Mission Bio Inc. , the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced the first-time deployment of one single-cell transduction assay across gene therapy company AVROBIO's lysosomal disorder pipeline. The two companies collaborated to develop a precise, reproducible assay using Mission Bio's Tapestri® Platform to rapidly measure the percentage of transduced cells in samples composed of thousands of cells. AVROBIO is presenting data today on the use of this assay to determine percent transduction in autologous, lentiviral vector-mediated, genetically modified drug product at the 4th Annual Gene Therapy Analytical Development (GTAD) Summit.

Transduction percentage is a critical quality attribute for cell and gene therapy manufacturing, measuring the successful transduction of therapeutic genes into target cells. Assessing this in thousands of cells with the previous percent transduction assay could take months, starting with a protracted cell growth step before performing PCR-based measurements on each single colony. But with the Tapestri®-based assay, AVROBIO's scientists can analyze thousands of single cells without a clonal outgrowth step, increasing throughput and precision.

In February 2022, Mission Bio transferred the new percent transduction assay to AVROBIO's contract research organization (CRO) partner for qualification and adoption in a current good manufacturing practice (cGMP) setting, with the aim of replacing the previous assay for lot release. AVROBIO expects to begin testing clinical samples with the new assay in early 2023 and include it as part of future regulatory filings, including Investigational New Drug (IND) submissions.

"The Tapestri®-based percent transduction assay has become an important tool providing us with the ability to rapidly and precisely measure critical quality attributes like percent transduced cells in drug product, and we believe this helps with demonstrating manufacturing capability and consistency," said Azadeh Golipour, Ph.D., Chief Technology Officer at AVROBIO. "Because we developed the assay agnostic to particular drug products, we can integrate it across multiple programs in our pipeline. We believe utilizing platform analytics across the entire portfolio reduces the chemistry, manufacturing, and controls (CMC) development timeline and allows us to deliver potentially life-changing therapies to patients with lysosomal disorders."

"The output of this collaboration with AVROBIO marks an important milestone, as we work to demonstrate the utility of single-cell multi-omic assays for therapeutic development and clinical testing," said Yan Zhang, Ph.D., CEO of Mission Bio. "Regulators and drug developers are demanding better ways of measuring various genomic attributes in a GMP environment to ensure safety and efficacy as cell and gene therapies progress through the clinic."

In a single high-throughput assay developed with the Tapestri® Platform, Mission Bio's biopharma partners can assess multiple characteristics of single gene-modified cells, including transduction efficiency, vector copy number, on- and off-target genome edits, zygosity, and aberrant translocations.

Dwayne Neal, Senior Director, Analytical Sciences & Technology, at AVROBIO, will share usage details and data on the assay in a talk titled "Single-Cell Genomics & NGS to Determine Percentage Transduction in Autologous Lentiviral Vector Drug Product." The presentation will be held in-person only as part of the GTAD Summit's Molecular Biology track on Nov. 29 at noon ET.

To learn more about Mission Bio and the Tapestri® Platform, please visit www.missionbio.com.

About Mission Bio

Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others.

The company's Tapestri® Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri® is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution. The Tapestri® Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide to develop treatments and eventually cures for cancer.

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