case-study

Case Study: PTC Therapeutics Unifies Small and Large Molecule Discovery with Sapio Lab Informatics Platform

Executive Summary

PTC Therapeutics, a global biopharmaceutical company, adopted Sapio’s unified, science-aware™ Lab Informatics platform to integrate its research approach and consolidate vendor solutions. This transition enabled PTC to streamline workflows for compound discovery, unify data capture and analysis for over 200 R&D scientists, and achieve end-to-end management of materials. By consolidating disparate informatics vendors onto a single, no-code configurable platform, PTC has simplified its user experience and established a robust foundation for adapting to evolving research needs and reducing costs.

The Customer: PTC Therapeutics

Company Overview

PTC Therapeutics was founded in 1998, focusing on leveraging expertise in RNA biology to discover and develop treatments for patients living with rare diseases. The company has deployed three biotechnology platforms: Bio-e (targeting oxidative stress and inflammation pathways), Nonsense suppression (bypassing mutations for inherited diseases), and Splicing modulation (reducing mutations causing human diseases). 

In its first 25 years, PTC has a commercial portfolio that includes six medicines treating Duchenne muscular dystrophy, spinal muscular atrophy, and several other rare genetic diseases.

Background

Like many established pharmaceutical and biotechnology companies, PTC had accumulated an array of software to support its researchers, including sample management and compound registration systems, lab automation systems, and data visualization and reporting tools.

The Challenge: Data Fragmentation and Inefficient Workflows

PTC’s existing tools, while well-liked by the team, were problematic from a research informatics perspective. One-off solutions might handle a specific workflow well, but data from those solutions often couldn’t be accessed easily by other scientists using their preferred solutions. The resulting data silos complicated workflows and slowed decision-making.

Additionally, some scientists relied on cobbled-together, homegrown solutions, such as Excel spreadsheets, to manage data. For instance, many individual teams maintained separate spreadsheets to track materials being used in experiments. Plus, some vital information on experiments run at PTC wasn’t being digitized; most scientists at PTC still documented their work in paper lab notebooks.

The Solution: Sapio’s Unified Informatics Platform

Why Sapio Sciences Was Chosen

PTC reviewed an extensive list of potential vendors and spoke to other pharmaceutical and biotechnology companies about their preferred vendor solutions. PTC ultimately selected the Sapio Sciences unified lab informatics platform, which offers a genuinely configurable, no-code/low-code, all-in-one platform essential to PTC’s research informatics team requirements.

• A complete solution that would provide researchers with easy access to the research data needed to support their daily tasks and effectively collaborate with their peers.
• A genuine platform delivering templated workflows and processes out of the box, with the ability to add functionality without complex coding.
• The ability to configure the platform to individual user roles, such as turning functionality on and off or setting permissions to access data.
• The capability to take full control over the solution configuration, rather than having to rely on vendor professional services.

Implementation Overview

PTC has opted for a phased implementation of the Sapio platform, focusing initially on rolling out the ELN functionality for managing materials and inventory. “With materials management we can realize immediate cost savings with the Sapio platform because it feeds into many downstream processes. For example, by simply helping scientists know where materials currently reside, they can avoid purchasing the same items over and over,” said Joseph Tivade, Senior Analyst at PTC Therapeutics.

Key Features

PTC adopted Sapio’s all-in-one laboratory information management system (Sapio LIMS), electronic lab notebook (Sapio ELN), and scientific data management platform. The unified platform has enabled PTC to:

• Unified, Science-Aware™ Platform: Consolidates LIMS, ELN, and SDMS into a single enterprise solution.
• No-Code Configurable Platform: Simplifies the user experience and allows for rapid adaptation and adding functionality without complex coding.
• Streamlined Workflows: For compound discovery, rapidly adaptable to evolving research needs.
• Unified Data Capture, Access, and Analysis: Fosters better collaboration and decision-making among PTC’s team of more than 200 R&D scientists.
• End-to-End Materials Management: Covers initial request to storage, locations, movement, inventory, and replenishment.
• Templated Workflows: Delivered out-of-the-box.
• Role-Based Configuration: Ability to configure functionality and permissions based on individual user roles.

The Results: Streamlined Operations, Cost Savings, and Enhanced Adaptability

The Sapio platform has enabled PTC to integrate its approach to research with a single enterprise platform and consolidate vendor solutions.

Immediate Cost Savings and Optimized Materials Management

Joseph Tivade, Senior Analyst at PTC Therapeutics, highlighted that “With materials management we can realize immediate cost savings with the Sapio platform because it feeds into many downstream processes. For example, by simply helping scientists know where materials currently reside, they can avoid purchasing the same items over and over.” This is achieved through end-to-end management of materials, covering initial request to storage, locations, movement, inventory, and replenishment.

Enhanced Operational Control and Efficiency

The PTC research informatics team now has complete control over the solution configuration. This has streamlined workflows for compound discovery and allowed them to rapidly adapt to evolving research needs. Furthermore, unified data capture, access, and analysis foster better collaboration and decision-making among PTC’s team of more than 200 R&D scientists.

Future-Ready and Scalable Research Infrastructure

By consolidating disparate informatics vendors onto a single, no-code configurable platform, PTC has established a robust and adaptable infrastructure. Joseph Tivade emphasized this, stating, “Through a combination of Sapio’s out-of-the-box workflows, no-code configurability, and some custom services, we’ve built a solution that can support our research data processes now and grow with us as we consider how to, for instance, feed research data into machine learning.”

Customer Quotes

“With materials management we can realize immediate cost savings with the Sapio platform because it feeds into many downstream processes. For example, by simply helping scientists know where materials currently reside, they can avoid purchasing the same items over and over.” – Joseph Tivade, Senior Analyst at PTC Therapeutics

“Through a combination of Sapio’s out-of-the-box workflows, no-code configurability, and some custom services, we’ve built a solution that can support our research data processes now and grow with us as we consider how to, for instance, feed research data into machine learning.” – Joseph Tivade, Senior Analyst at PTC Therapeutics

Lessons for Biopharmaceutical Companies

This case study offers critical insights for other established pharmaceutical and biotechnology companies seeking to optimize their research informatics:

• Prioritize Unified Informatics Platforms: Consolidate disparate software solutions onto a single, integrated LIMS/ELN/SDMS platform to eliminate data silos, simplify the user experience, and reduce overall costs.
• Leverage No-Code/Low-Code Configurability: Opt for platforms that empower in-house teams to rapidly adapt workflows, add functionality, and configure solutions to specific user roles without extensive custom coding or heavy reliance on vendor professional services.
• Implement Robust Materials Management: Focus on end-to-end materials tracking, from request to replenishment, to realize immediate cost savings by optimizing inventory and preventing redundant purchases.
• Foster Data Unification for Collaboration: Choose solutions that unify data capture, access, and analysis across large R&D teams to enhance collaboration and accelerate decision-making in complex discovery processes.

• Build for Future Adaptability: Select a platform with templated workflows and the inherent flexibility to support evolving research needs, including the integration of new processes like small molecule discovery and future technologies such as machine learning.

Conclusion

PTC Therapeutics has successfully transformed its approach to research by adopting Sapio’s unified, science-aware™ lab informatics platform. This has enabled the company to bridge small and large molecule discovery, streamline workflows, unify data, and consolidate vendor solutions. By leveraging Sapio’s configurable LIMS/ELN, PTC has not only simplified its operations and reduced costs but also established a robust, adaptable enterprise platform that will support its evolving research needs and drive future advancements in rare disease therapies.

Download the Case Study

FAQ

Q: Why did PTC Therapeutics choose Sapio?
A: To consolidate disparate informatics systems into a unified, no-code configurable platform that supports both small and large molecule discovery.

Q: What benefits did the Sapio platform deliver?
A: Streamlined workflows, unified data access for over 200 R&D scientists, and end-to-end materials management.

Q: What makes the Sapio platform unique for PTC?
A: Its no-code/low-code adaptability, role-based configuration, and out-of-the-box templated workflows.

Q: What were the measurable outcomes?
A: Immediate cost savings, simplified inventory control, and a scalable foundation for future AI integration.