Cell and Gene Therapy Developers Are Worried About Clinical Holds From Inadequate Early Product Characterization, According to Mission Bio Survey
28 Feb 2023
Gene Therapy
Respondents to the survey, which was administered via third party, point to lengthy protocols and lack of certainty in determining critical quality attributes among their top concerns
SOUTH SAN FRANCISCO, Calif., Feb. 28, 2023 /PRNewswire/ -- Mission Bio, the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced results of a new survey revealing persistent concerns by cell and gene therapy (CGT) developers over significant challenges to product characterization, despite rapid progress in the field. The survey of scientists in the space, deployed through RegMedNet in June 2022, uncovered key challenges and workflow limitations related to their analytical characterization process, which is a pivotal step in establishing measures of quality, potency, and potential safety in this novel class of therapies.
In the survey, Mission Bio found that the vast majority of respondents require multiple assays and instruments but rely on limited drug samples to complete their characterization assays. Respondents indicated that their top pain point was the length of time it takes (three or more weeks) to complete their analytical characterization assays, supporting the need for faster assays that can expedite drug development and delivery to patients. Additionally, one-third of respondents felt their current analytical approach does not adequately determine or measure critical quality attributes (CQAs), which are crucial for ensuring that the quality of the drug product is high enough for use by patients.
The survey consisted of an online questionnaire that was answered by 200 cell and gene therapy developers who include analytical characterization as part of their job responsibilities, excluding Mission Bio customers. The respondents represented CGT developers globally, and across pharmaceutical, academic, and contract research organization (CRO) settings.
Many of the issues uncovered by the survey suggest a limitation in analytical assays to provide high-resolution information in short time frames for therapies that are inherently complex. To address these challenges head-on, Mission Bio has worked with the CGT community-at-large to develop solutions using its single-cell DNA and multi-omics Tapestri® Platform. Tapestri® consolidates and accelerates analytical characterization workflows that historically have required weeks using multiple instruments into a matter of days, while delivering actionable high-resolution single-cell data. CGT developers like AVROBIO and CROs like Avance Biosciences have already begun deploying Tapestri® assays to characterize clinical batches of gene modified cell-based therapies.
"This survey is the latest reflection of Mission Bio's commitment to ensuring the success of emerging technologies," said Vanee Pho-Conners, Senior Director of Product Management at Mission Bio. "It extends beyond our products, through our work across the industry in collaborative bodies like the National Institute of Standards and Technology (NIST) Genome Editing Consortium, to increase confidence in their usage and lower potential risks in drug products."
"More rapid and reliable methods to analyze cell and gene therapies is mission critical for drug developers, and the Tapestri® Platform provides a groundbreaking solution," said Chris Mason, Professor of Cell & Gene Therapy at University College London and Mission Bio Scientific Advisory Board member. "For the first time, we are able to measure multiple therapeutic parameters simultaneously, such as vector copy number, transduction efficiency, gene editing outcomes, and genomic stability. And we can do it in a matter of days, minimizing sample usage and accelerating data insights that may provide critical indicators of safety and potency. The Tapestri® Platform's ability to provide these analytics in a consistent, high-resolution manner at the single-cell level, while eliminating laborious steps, addresses the industry's top concerns."
Access the full survey report here: https://go.missionbio.com/cgt-survey-report
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.
CONTACT: Will Walton, [email protected]
SOURCE Mission Bio
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