Applying a patient-driven strategy to treat genetically defined diseases
Launched from Third Rock Ventures, Fulcrum Therapeutics went public in 2019 by leveraging its proprietary technology, libraries, and databases to fuel its Product Engine.
Fulcrum Therapeutics focuses on treating genetically defined rare diseases, such as facioscapulohumeral muscular dystrophy (FSHD) and sickle cell anemia, by modulating expression of disease-relevant, root cause genes.
From using patient samples for drug discovery to using patient data to identify targets, Fulcrum Therapeutics has placed patients at the center of their pipeline decisions. The company’s R&D teams must work across different disease areas and access a wide variety of models, assays, technologies, and expertise from external providers.
Increasing speed to market to bring value to patients and investors
As a lean biotech with ambitious goals, Fulcrum Therapeutics needed to maximize R&D speed and productivity. Given the highly competitive environment, speed was essential for filing and protecting IP, as well as for quickly moving potential therapies into clinical development.
- Meeting aggressive R&D timelines: Fulcrum Therapeutics advanced Losmapimod, a potential treatment for a form of muscular dystrophy into Phase 2 clinical trials by year two post-launch, and recently initiated a Phase 1 trial in adult healthy volunteers with FTX-6058 for Sickle Cell Disease.
- Overcoming delays related to project initiation: Given their dependence on networked R&D, biotech companies such as Fulcrum Therapeutics need to establish contracts with providers, negotiate discounts, and build IT infrastructure to manage external R&D. Traditionally, these steps can slow down drug discovery.
- Provider discovery: It is challenging for R&D scientists to spend time discovering and building relationships with service providers that are qualified, available, and cost-effective.
- Mitigating risk: To maintain their agility, Fulcrum Therapeutics needs to outsource key projects and minimize administrative overhead while managing the IP, confidentiality, compliance, and budgetary risks associated with outsourcing R&D.
A technology platform built to accelerate biotech R&D
The Science Exchange SaaS-enabled marketplace was designed to solve the project initiation challenges for emerging biotech R&D organizations. By eliminating the need and time delay to establish one-off provider contracts and by integrating directly with existing IT infrastructure, Science Exchange enables biotech companies to focus immediately on execution.
Fulcrum Therapeutics was able to execute its innovation strategy for sickle cell disease as Science Exchange provided the team rapid access to mission-critical services. For this discovery research program, Fulcrum Therapeutics had been trying for over a year to find a source of specific blood cells from these patients. Through Science Exchange, they were able to search for and identify a provider for whole blood collection (Figure 1) and another provider who could perform the required cell isolation, with seamless sample transfer in between. No additional contracts were needed prior to starting the work, as Science Exchange’s globally recognized standard MSA was already in place.
Fulcrum Therapeutics adopted the Science Exchange platform to source, manage, order, and track their outsourced R&D projects and were immediately able to:
- Increase speed of drug discovery by initiating R&D services from prequalified, fully contracted providers
- Maximize buying power by leveraging Science Exchange’s pre-negotiated discounts with providers of key services.
- Obtain strategic insights and business intelligence, and streamline internal communication by using transparent, real-time reporting to track external R&D spend, including monitoring of blanket POs.
- Boost operational efficiency by integrating Science Exchange with Fulcrum Therapeutics’ single sign-on, ERP, and file storage systems, creating a secure, efficiently connected R&D workflow.
Freedom and productivity for Fulcrum Therapeutics’ scientists
Thanks to the Science Exchange centralized, transparent reporting features, R&D leaders at Fulcrum Therapeutics gained real-time visibility into external R&D projects. Researchers were also freed from all administrative tasks associated with searching for and qualifying R&D service providers. They saved hundreds of hours collectively and were able to focus instead on planning experiments, analyzing data, and communicating results.