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Big 2024 Plans: Building the First Multimodal R&D Platform
Krystal Biotech is changing the way rare diseases are treated. Their corrective and redosable gene therapies deliver new hope to patients who have long been told their situations were hopeless. The company’s first approved product—VYJUVEK™ (beremagene geperpavec)—is used to treat dystrophic epidermolysis bullosa, an incurable, potentially-fatal, skin-blistering condition. VYJUVEK™ was developed using Krystal’s patented STAR-D Gene Therapy Platform. STAR-D leverages modified type-1 herpes simplex virus, which can penetrate a broad range of skin cells to introduce therapeutic genes that trigger functional protein production, all without integrating into the patients’ native genetic materials or virally replicating.
Krystal’s R&D teams are applying this novel STAR-D Gene Therapy Platform to develop a number of other rare-disease gene therapies for skin diseases and beyond. They are also exploring whether STAR-D could help in developing treatments for more prevalent, chronic conditions. The company has a robust pipeline spanning dermatologic, respiratory, ophthalmologic, and oncologic disease.
With ambitious goals for STAR-D, Krsytal leadership knew that fully capturing their experimental work and building off their knowledge was essential; they also realized that their existing informatics set-up wasn’t ideal for supporting their innovation and growth. Chelsea Geruschat, Senior Research Associate in Downstream Process Development at Krystal Biotech, explained, “There was a lot of flexibility in our previous system, and while some flexibility in data management is essential, we worried too much could impact our ability to record and organize our experiments and results. Additionally, when data was siloed in large Excel files, efficient retroactive data analysis was becoming a challenge.”
Krystal Biotech’s R&D Data-Management Goals
Krystal sought a new R&D informatics solution that would satisfy key goals around data management, including:
Data Capture - Quick and clean data capture via more formalized procedures.
Data Format and Storage - Standardized and cross-checked data united in a centralized data lake, not scattered across disconnected data silos.
Data Access - Fast scientific search to find all relevant data, no matter how new or old.
Data Sharing - Easier data sharing across groups, with the ability to create relationships between experiments.
Data Security - Permission controls to help keep data equally accessible and secure.
Future-Proof R&D Data Management
Krystal’s R&D leaders set out to find a solution that would help them quickly achieve their data-management goals. The company believed that finding the right solution would free its researchers to focus their time on innovating, not data wrangling. R&D leaders set a goal to find an ELN that would best help them manage their experimental data in the near-term. But, they also kept in mind that, in the long-term, they would likely need more robust capabilities across biology and chemistry, such as specialty science applications, inventory and registration systems, assay management, and statistics and visualization software.
Because many team members had been let down by other ELN and informatics solutions in the past, there was a bit of trepidation and worry that it might be impossible to find an informatics solution with the power and flexibility needed to support Krystal’s ambitious and complex R&D programs. But Dotmatics is different.
Dotmatics’ Flexible and Scalable R&D Platform and ELN
Dotmatics Platform and ELN could help Krystal in both the near and long term. Dotmatics offered the best of both worlds. In the short term, Dotmatics ELN’s flexibility and user friendliness meant Krystal’s R&D teams could nimbly shift to new processes to optimally capture and manage their experimental data. In the long-term, Dotmatics Platform’s rich collection of available scientific capabilities could support Krystal’s changing R&D needs as teams grew and programs evolved.
Erik Schneider, Senior Manager of Downstream Process Development and Validation at Krystal Biotech, explained, “In the end, the Dotmatics Platform felt like it would be easy to immediately pick up, while also offering more advanced capabilities. We liked that we could first adopt the basic ELN tool and expand from there. While we wanted to get up and running quickly, it was still important that we have the option to add new tools and functionality down the line, when we get to the point when we need them.”
Geruschat echoed that sentiment, explaining, “With other ELNs I have used in the past, it always felt like we hit a wall. We would think, ‘This is all the ELN can do for us.’ With the Dotmatics Platform, we definitely haven’t hit a wall, and I don’t foresee that happening any time soon.”
With Dotmatics, Krystal now has a user-friendly, secure, and scalable R&D informatics infrastructure. Krystal’s R&D data are now standardized, well-organized, and easy-to-access, freeing researchers to extract valuable insights and realize the true potential of their STAR-D Gene Therapy Platform.
