What to Look for in an Informatics Platform for Gene Therapy R&D

As the fastest growing class of biopharmaceuticals, gene therapy marks a new era of medicine, bringing with it a new paradigm for R&D where the product is a complex combination of genetic materials and viral or non-viral delivery vectors. As a result, conventional lab informatics platforms are not always optimal for gene therapy R&D.

Gene therapy development—from early stage research to discovery through commercialization—requires careful orchestration of complex processes and cross-functional teams. Regardless of the scientific approach, from gene editing to viral vectors, several teams need to work closely together to support the entire workflow; generate, analyze, and transfer massive amounts of data; and ultimately get life changing therapies to patients.

Having worked with hundreds of gene therapy companies, Benchling knows what it takes to be successful in this competitive market. To help you get ahead, we’ve put together a Buyer's Guide with the key criteria for evaluating an informatics solution for gene therapy R&D and included a few highlights below.

Read the buyer's guide

Informatics Platforms for Gene Therapy R&D: Current Status and Gaps

Gene therapy companies—both small and large—need an informatics platform that can help them address the complexities of vector design, process optimization, product characterization, and data handoffs.

R&D teams need to be able to:

• Fully map and interconnect samples with sequence-level data and experimental results

• Design optimal vectors by leveraging institutional knowledge and experience

• Optimize complex R&D processes from target identification through scale up

• Efficiently collaborate and transfer data between research, development, and analytical teams

However, most available software solutions fall short because they are unable to handle the diversity of entities (e.g., DNA, plasmids, endonucleases, guide RNAs, etc.), are difficult to configure to the specific needs of both research and development teams, and are unable to adapt to the rapidly changing science while continuing to support current therapies in the pipeline.

Because of these limitations, gene therapy companies end up relying on multiple, disconnected solutions, such as laboratory information management systems (LIMS), electronic lab notebooks (ELNs), paper notebooks, and spreadsheets—creating siloed, difficult-to-trace data, which ultimately results in missed opportunities and slows progress to market.

The ideal informatics platform will offer flexibility to power ever-changing research initiatives as well as structure to facilitate standardization for robust gene therapy development.

Informatics Platforms for Gene Therapy R&D: Evaluation Criteria

Getting the right software solution as early as possible is imperative for gene therapy companies, as it can be the difference between efficient and speedy development and wasted time and resources.

When evaluating an informatics platform, ask yourself these four questions:

Does it support your science?

Research and development teams require different characteristics in a software solution. Research teams work with DNA, plasmids, vectors, guide RNAs, endonucleases, and numerous other entities that are designed and tested, creating an abundance of data that needs to be mapped and analyzed. Gene therapy development teams need to be able to create, optimize, and monitor standardized bioprocessing, purification, and product characterization workflows to ensure consistency and quality between batches.

Will it improve productivity?

Minimizing manual labor, automating repetitive tasks (e.g. plasmid construction and gRNA design), and reducing errors are a must when it comes to boosting productivity. An ideal solution has easy-to-use, built-in features that help teams accomplish this.

Will it help your teams collaborate?

Gene therapy R&D requires many teams that need to work closely together to push a candidate through the pipeline. Suitable software solutions for gene therapy R&D automatically link samples with associated assay data, enabling you to get full experimental context on a sample even if others have done the work and making it easy for team members to work on different aspects of a project or experiment simultaneously.

Does it offer access to insights?

The ideal informatics platform connects samples, results data, process parameters, and more and offers a variety of ways to visualize information so teams can analyze data in new ways to find critical insights that drive decision making.

So how can Benchling help? For a vector core scientist, we can help increase the efficiency and accuracy of plasmid design. For a process engineer, we can help centralize and optimize production processes. For analytical scientists, we can simplify data and sample handoffs between teams.

“With Benchling, our speed has doubled, communication has improved exponentially, and it’s decreased scientist frustration level beyond measure.”

– Brenda Minesinger, Principal Scientist, Intellia Therapeutics

Informatics Platforms for Gene Therapy R&D: The Ideal Solution

The ideal informatics platform for gene therapy companies will go beyond the capabilities of conventional LIMS and ELNs by supporting the entire R&D lifecycle end-to-end, by being easy to configure and user-friendly, and by being able to adapt to the rapidly evolving needs of gene therapy and gene editing R&D. This enables biopharmaceutical organizations to accelerate gene therapy R&D and bring more breakthrough therapies to market faster.

“It feels good to have something automated with just a single click, where before it took half an hour. As more people are getting familiar with [Benchling], we’re saving even more time.”

– Tim Hoogervorst, Technician, uniQure