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CASE STUDY

uniQure: Bringing a gene therapy pioneer from paper to the cloud

Illustration by David S. Goodsell. doi: 10.2210/rcsb_pdb/goodsell-gallery-004

A global leader in gene therapy

Having originated the first approved gene therapy in 2012, uniQure is a leading pioneer in the field. 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.

Company

Number of employees: 250 – 1000

Industry: Biopharma

Location: Amsterdam, Netherlands

Goal

Bring a gene therapy pioneer from paper to the cloud.

CHALLENGES

Difficulty tracking experiments with paper documentation
Paper-based workflows made it difficult to track results and streamline experiment execution.

Sharing data and collaborating across international sites
As an international company with worksites in Amsterdam and Lexington, MA, uniQure found it challenging to share data and collaborate across research sites.

Difficulty assessing experiment quality
A lack of standardized Notebook templates led to nonstandard experimental workflows and impeded quality control.

RESULTS

End-to-end tracking for gene therapy workflows
With Benchling, scientists can track from vectors, to tissue samples, to viral batches and experimental results.

International collaboration — even while working remotely during the COVID-19 pandemic
Having a digital system enables internationally distributed researchers to collaborate and discover data much more easily – even when working from home due to the COVID-19 pandemic.

Improved efficiency and enthusiastic adoption
Benchling Notebook templates massively improve scientist efficiency. 100% of the scientists licensed to use Benchling use the Notebook every day.

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