The Business of Biofabrication

Overview

On June 24, 2025, United Therapeutics Corporation (Nasdaq: UTHR) and Intermountain Health treated the first patient in a Phase 1 clinical trial of miroliverELAP®, an external liver support system that leverages a bioengineered porcine scaffold repopulated with human cells. This landmark event marks the world’s first human use of a bioengineered organ alternative designed for acute liver failure (ALF). Below, we assess the technology, strategic partnership, market opportunity, competitive landscape, and outlook for biofabricated organ products.

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Technology Platform

• Core Components
  • Scaffold: Decellularized porcine liver (MIRO-001) providing native extracellular matrix architecture
  • Recellularization: Human endothelial and hepatocyte cells seeded to restore metabolic and vascular functions
  • Extracorporeal Circuit: Single-use bioreactor system that perfuses patient blood through the bioengineered liver
• Advantages
  • Temporary bridge for patients ineligible for transplantation
  • Potential to reduce mortality (~30% without transplant) and alleviate donor organ shortages
  • Modular design that could extend to other organ systems (hearts, kidneys, lungs)

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Strategic Partnership

• United Therapeutics / Miromatrix
  • Established capabilities in xenotransplantation and regenerative medicine platforms
  • Vertical integration in organ alternative manufacturing
• Intermountain Health
  • Top U.S. nonprofit health system with robust clinical trial infrastructure
  • Early adopter of cutting-edge therapeutics, enhancing trial enrollment and operational excellence

Insight: Aligning a therapeutics developer with a large integrated health system accelerates patient access, regulatory feedback, and real-world data generation—critical for de-risking subsequent trial phases.

Technology Overview

Developed by Miromatrix Medical (a United Therapeutics subsidiary), miroliverELAP consists of a decellularized porcine liver scaffold (MIRO-001) that has been repopulated with human endothelial and hepatocyte cells. Through an extracorporeal blood circuit, the system temporarily takes on critical liver functions, offering support where donor organs are unavailable or contraindicated.

Strategic Partnership

This collaboration between United Therapeutics and Intermountain Health leverages the strengths of both organizations. United Therapeutics brings deep expertise in organ-alternative manufacturing, having built integrated xenotransplantation and allogeneic regenerative medicine platforms spanning hearts, kidneys, livers, and lungs. Intermountain Health, one of the nation’s premier nonprofit health systems, contributes extensive clinical-trial infrastructure and a strong track record of adopting cutting-edge therapies. Together, they accelerate trial enrollment, operational execution, and real-world data generation.

Unmet Clinical Need

The unmet need in acute liver failure (ALF) is substantial: approximately 86,000 U.S. hospitalizations occur annually for ALF and related conditions, with roughly 30 percent of patients dying without a transplant. Current care paradigms—comprising intensive supportive care, transplantation, or spontaneous recovery—leave a significant survival gap for those who cannot receive donor organs. By functioning as a temporary bridge, miroliverELAP has the potential to reduce mortality, shorten ICU stays, and mitigate complications such as cerebral edema or bleeding.

Competitive Landscape

In the competitive landscape of liver-support technologies, several groups explore enzyme-based or spheroid-based devices. However, miroliverELAP’s use of a whole-organ scaffold combined with human cells more closely mimics native liver architecture and function than microtissue systems. This biomimetic advantage could translate into more robust metabolic support and improved patient outcomes, differentiating it from earlier preclinical efforts.

Regulatory Pathway

Regulatory progress has been carefully staged: a Q-Submission clarified potential safety and effectiveness questions, and the ongoing Phase 1 trial across eight U.S. sites will assess safety and initial mechanistic biomarkers. If safety is confirmed and early efficacy signals emerge, a Phase 2 study could commence by 2027. Concurrently, United Therapeutics is engaging with CMS to explore reimbursement pathways—including pass-through payments for device–biologic combination products—to facilitate broader adoption.

Manufacturing & Cost Considerations

Looking ahead, scaling the manufacturing process will be paramount. Achieving consistent decellularization and recellularization at commercial volumes requires significant bioprocess engineering and GMP capacity. Cost management will hinge on optimizing porcine scaffold sourcing, human cell procurement, and single-use perfusion cartridge production.

Pipeline Expansion

Success in liver support could validate the underlying biofabrication platform for other organs—potentially creating a multi-organ pipeline that addresses several end-stage organ diseases. The ability to replicate this approach for hearts, kidneys, and lungs would represent a profound shift in how organ failure is managed.

Conclusion

The first human use of miroliverELAP marks a pivotal moment in the evolution of biofabricated organ therapies. By combining a biologically faithful scaffold with human cells and leveraging the capabilities of a leading health system, United Therapeutics and Intermountain Health have set the stage for transforming the treatment landscape for acute liver failure—and, potentially, many other organ-failure indications in the years to come.

About United Therapeutics

United Therapeutics Corporation (Nasdaq: UTHR) is a public-benefit biotechnology company committed to creating novel therapies for life-threatening conditions. With active programs in small-molecule drugs, biologics, and organ-manufacturing platforms, United Therapeutics leverages xenotransplantation, allogeneic, and autologous regenerative medicine approaches to address critical organ shortages. The company’s long-term vision is to make manufactured organ alternatives widely available, transforming the standard of care for end-stage organ disease.

About Miromatrix

Miromatrix Medical Inc. is a wholly-owned subsidiary of United Therapeutics focused exclusively on bioengineering whole-organ scaffolds. By decellularizing animal organs and recellularizing them with human cells, Miromatrix develops off-the-shelf organ models capable of performing essential physiological functions. Its lead program, miroliverELAP®, represents the first externally perfused, bioengineered liver being tested in humans, setting the stage for future organ-alternative products.

About Intermountain Health

Intermountain Health is a nonprofit integrated health system serving communities across Utah, Idaho, and Nevada. Consistently recognized for clinical innovation and quality outcomes, Intermountain operates more than 30 hospitals and 400 clinics. Its research arm and centralized clinical-trial infrastructure enable rapid adoption and rigorous evaluation of emerging therapies. As a partner in the miroliverELAP® trial, Intermountain Health provides the operational excellence and patient-care expertise necessary to advance next-generation organ-support technologies.

For more info: https://www.unither.com/

Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include expectations regarding the continued enrollment, safety assessments, and potential expansion of the miroliverELAP® Phase 1 trial, as well as plans to engage with regulatory and reimbursement agencies to support commercialization. Actual outcomes may differ due to risks such as manufacturing scale-up challenges, regulatory approval uncertainties, and evolving clinical trial data.