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Case Studies > Bringing a gene therapy pioneer from paper to the cloud

Bringing a gene therapy pioneer from paper to the cloud

Technology Category
  • Infrastructure as a Service (IaaS) - Cloud Computing
  • Platform as a Service (PaaS) - Data Management Platforms
Applicable Industries
  • Healthcare & Hospitals
  • Life Sciences
Applicable Functions
  • Product Research & Development
  • Quality Assurance
Use Cases
  • Collaborative Robotics
  • Predictive Maintenance
Services
  • Cloud Planning, Design & Implementation Services
  • Data Science Services
The Challenge
uniQure, a leading pioneer in gene therapy, faced several challenges in their operations. As an international company with worksites in Amsterdam and Lexington, Massachusetts, they found it challenging to share data and collaborate across research sites. Their paper-based workflows made it difficult to track results and streamline experiment execution. A lack of standardized Notebook templates led to nonstandard experimental workflows and impeded quality control. These challenges were further exacerbated by the COVID-19 pandemic, which forced some uniQure scientists to work from home.
About The Customer
uniQure is a leading pioneer in the field of gene therapy. The company originated the first approved gene therapy in 2012. By leveraging their modular and validated technology platform, uniQure is rapidly advancing a pipeline of AAV-based gene therapies to treat patients with hemophilia, Huntington’s disease, and other severe genetic diseases. The company is international with worksites in Amsterdam and Lexington, Massachusetts. It employs between 250 to 1000 employees. The teams using Benchling within uniQure include Research, Analytical development, Nonclinical, Vector development, and Process development.
The Solution
uniQure implemented Benchling's solutions to overcome their challenges. Benchling's Notebook, Registry, and Inventory applications were used to create a complete history of every experiment, enabling scientists to track from vectors, to tissue samples, to viral batches and experimental results. Benchling protocols standardized procedures and data entry, making it easier for scientists to analyze their experiments. The company's analytical and product development teams used Benchling to track every sample in their freezers, linking location data directly to results readouts. This digital system enabled internationally distributed researchers to collaborate and discover data much more easily, even when working from home due to the COVID-19 pandemic.
Operational Impact
  • End-to-end tracking for gene therapy workflows was achieved, with Benchling automatically creating a complete history of every experiment.
  • International collaboration was improved, even while working remotely, due to the digital system.
  • Efficiency was improved and enthusiastic adoption was seen, with 100% of the scientists licensed to use Benchling using the Notebook every day.
Quantitative Benefit
  • 100% of the scientists licensed to use Benchling use the Notebook every day.

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