Encoded Therapeutics社、Genedata Bioprocessを導入：AAVによる遺伝子治療の開発を加速

Genedata Bioprocess streamlines gene therapy development processes and drive decision-making across Encoded’s R&D organization

June 30, 2021
Basel, Switzerland and South San Francisco, CA, USA

Genedata, the leading provider of enterprise software solutions for biopharmaceutical R&D, today announced that Encoded Therapeutics, a biotech company focused on developing precision gene therapies for pediatric central nervous system (CNS) disorders, has chosen Genedata Bioprocess^® to digitalize their proprietary gene therapy R&D technology. The Genedata platform will serve as the digital backbone to further advance Encoded’s approach of synthetically engineering regulatory sequences to modulate or create desired patterns of gene expression.

“Genedata Bioprocess has an excellent reputation as a leader in biologics process development software,” said Martin Moorhead, Ph.D., Chief Technology Officer at Encoded. “We chose Genedata because our teams needed a central, scalable, and robust workflow platform to first collect and track all data across our entire process development organization, and then trace and interpret the data in real-time, facilitating more informed decision-making. Leveraging Genedata’s capabilities will enable us to continue scaling Encoded’s process development capabilities to drive growth and value in gene therapy.”

The need to tie analytical results together from all process development operations has become a bottleneck in the gene therapy sector, as the amount of data to manage continues to exponentially increase. Genedata Bioprocess enables full traceability of R&D data while concurrently ensuring data integrity around all molecules, samples and assay, and analytics data. The Genedata platform also automates processes, integrates instruments, and prints barcoded labels, forgoing the need to manually apply IDs or run numbers to samples, resulting in further efficiencies for R&D teams.

Encoded’s cell-selective approach may overcome selectivity and potency limitations of first-generation adeno-associated virus (AAV)-mediated gene therapies. By leveraging non-coding DNA to recapitulate natural patterns of gene expression, the company is creating precision gene therapies to target specific cell types or levels of expression. Encoded is independently building a wholly owned pipeline focused on pediatric CNS disorders. The company’s lead program, ETX101, is designed to address the underlying cause of SCN1A+ Dravet syndrome as a potential first-in-class, one-time gene therapy.

“While cell and gene therapies are dramatically changing the scope of diseases treatable today, the abundance and complexity of data collected for developing these breakthrough therapies are creating serious process and data management challenges,” said Othmar Pfannes, Ph.D., CEO of Genedata. “We are proud that Encoded, a cutting-edge biotech company in the cell and gene therapy space, has selected Genedata to fully collect, track, and manage the complex and diverse data and processes needed to develop gene therapy-based biotherapeutics. By digitalizing biopharma R&D workflows and accelerating decision-making, our platforms ultimately help bring these game-changing therapies faster to patients.”