Enabling a New Era of Advanced Therapeutics at UPMC

UPMC Enterprises is playing a critical role in the research and development of next-generation treatments across the health system and in Pittsburgh.

One of the exciting advances in health care treatment modalities to emerge from decades of basic science research and clinical trials is the rapidly expanding array of advanced therapeutics, a group of transformative therapeutic approaches designed to treat a variety of complex, chronic, or genetically driven conditions.

From made-to-order radiopharmaceuticals and customized cell-based immunotherapies to personalized gene modification techniques, novel surgical innovations, and breakthrough pharmacologic agents, these emerging and expanding interventions are beginning to deliver unprecedented health outcomes for patients with severe, previously untreatable, or intractable conditions.

However, these advanced next-generation therapeutic platforms and delivery systems present substantial challenges related to the criteria for optimal patient selection, manufacturing complexity, supply chain and delivery logistics, regulatory oversight and approvals, and ultimately cost, raising questions of how the health care system can ensure appropriate access to these breakthroughs.

UPMC Enterprises, the innovation, commercialization, and venture capital arm of UPMC, is playing a critical role in enabling the creation of next-generation therapeutics. The organization is investing in high-impact translational science and creating operational and strategic partnerships in the biotech space. It also is drawing on the immense talent pool at UPMC and the University of Pittsburgh, and externally in promising startups and mature companies alike, to support the development, testing, and integration of advanced therapeutics into clinical care and research.

This article explores UPMC Enterprises and the greater UPMC system’s role in building real-world delivery pathways for some of the most advanced and often logistically demanding treatment approaches to complex medical conditions. It examines the rationale behind these efforts and their practical implications for access, equity, and scalability, and outlines concrete examples currently in development and what may be possible in the future.

“The barriers can be significant, but the opportunity to change what we can offer patients drives us to keep pushing forward,” said Timothy Billiar, MD, Chief Scientific Officer and Executive Vice President at UPMC and Associate Senior Vice Chancellor for Clinical Academics at the University of Pittsburgh. Dr. Billiar provided perspectives on UPMC’s institutional philosophy for investing the financial and human capital resources necessary for bringing advanced therapeutic options to not only the systems’ own patient base but in expanding access broadly.

The Expanding Horizon of Therapeutics

The therapeutics era of medicine is entering a new phase of maturity — one driven by groundbreaking advances in immunology, molecular biology, genetics, and biomedical engineering. These scientific frontiers are reshaping what’s possible in the treatment of serious, chronic, and life-threatening conditions such as cancer, cardiovascular disease, genetic disorders, and autoimmune illnesses. 

At the heart of this transformation lies the extraordinary complexity of biological life at the genetic, cellular, and molecular levels. The diseases that emerge from these intricate systems are equally complex, demanding equally sophisticated and nuanced therapeutic strategies.

Today’s rapid technological innovation is accelerating our understanding of disease biology and unlocking entirely new therapeutic targets and modalities. These breakthroughs are enabling a fundamental shift — from one-size-fits-all treatments to personalized, precision medicine, and from symptomatic management to curative, disease-modifying therapies. This evolution is not just promising; it’s powerful. That is why UPMC is committed to investing in this space: the opportunity to change the course of disease and dramatically improve patient outcomes is both real and within reach. 

“We’re at a point where the technologies exist to dramatically reshape how we treat cancer, genetic disorders, and other serious diseases for many people,” said Dr. Billiar. “But every new avenue brings new responsibilities in how we evaluate safety, justify costs, and keep patient welfare at the center of it all. It’s not a question of why do it or do we want to do it, but one of how we do it.”

Building Radiopharmaceutical Infrastructure: The PharmaLogic Joint Venture

Radiopharmaceuticals are an increasingly important class of advanced therapeutics that combine targeted biological activity with the powerful therapeutic or diagnostic effect of radioactive isotopes. Their clinical utility has grown in oncology, where they can deliver potent, tumor-specific payloads while minimizing systemic toxicity and off-target effects.

However, radiopharmaceuticals often require on-site or near-site production due to the short half-lives of key isotopes, presenting a significant barrier to adoption outside major academic centers. In addition, unique instrumentation and radioactive shielding required to manufacture, handle, and administer radiopharmaceuticals adds to the infrastructure requirements for local manufacturing facilities, thus raising the barriers for health care systems to offer these transformational therapies.

To address these challenges, UPMC decided to partner instead of going it alone, and has entered a joint venture with PharmaLogic Holdings Corp. to build one of the largest radiopharmaceutical production facilities in the U.S. at UPMC Montefiore. The facility, currently in the final stages of construction, will support research, clinical trials, and commercial use of radioligand therapies by enabling a more predictable, efficient, and localized supply chain that is backstopped by the broader PharmaLogic network of sites in the U.S. The new facility at UPMC is expected to help integrate radiopharmaceuticals into everyday care, expand access to radiopharmaceuticals more broadly, and further establish UPMC as a leader in advanced diagnostics and therapeutics. 

“The decision to invest in this infrastructure was about making these therapies not only accessible but also clinically viable at scale,” said Dr. Billiar. “We need to build the systems that allow us to deliver the right compound to the right patient at the right time, especially when the logistics are this complex.”

Enabling Cell and Gene Therapy Through Biomanufacturing

Cell and gene therapies, ranging from CAR T-cell therapies for hematologic cancers to gene editing techniques targeting inherited diseases, continue to expand the therapeutic possibilities for complex and chronic conditions. Bringing these therapies to market requires more than promising science. It requires scalable, high-quality manufacturing technical expertise and a skilled workforce, along with the institutional infrastructure support, and supportive regulatory pathways.

The Pitt BioForge Biomanufacturing Center, a joint effort between the University of Pittsburgh and ElevateBio, was announced in 2022 and will anchor a new ecosystem for advanced biologics manufacturing in Pittsburgh. ElevateBio specializes in enabling technologies for next-generation therapies, including gene editing, induced pluripotent stem cell (iPSC) platforms, and vector and protein engineering. Through this partnership, ElevateBio will bring its integrated development and GMP manufacturing capabilities to Pittsburgh, significantly expanding the region’s capacity to support clinical-grade production of cellular and gene-based therapeutics. 

Funded in part by a $100 million grant from the Richard King Mellon Foundation, the Pitt BioForge is being developed to serve as a national resource for the production of cell and gene therapies. Locally, its creation will be a catalyst for regional economic development and clinical innovation around therapeutic R&D.

Several current UPMC Enterprises portfolio companies are expected to benefit from the infrastructure and capabilities that BioForge will provide. One company, BlueSphere Bio, is developing precision T cell receptor (TCR) therapies aimed at high-risk leukemia and solid tumors, using its TCXpress™ and NEOXpress™ platforms.

Another UPMC Enterprises portfolio company, Avista Therapeutics, founded by University of Pittsburgh Opthalmology researcher Leah Byrne, PhD, is focused on developing next-generation adeno-associated virus (AAV) vectors for ophthalmologic gene therapy.  

Access to regional biomanufacturing capacity such that will exist at Pitt BioForge will reduce barriers to scale-up and speed the path from bench to bedside for these and other therapeutic options in the development pipeline.

Advancing TIL Therapy and Personalized Immunotherapy

UPMC is also home to one of the nation’s few tumor-infiltrating lymphocyte (TIL) therapy programs. Led by Udai Kammula, MD, director of the Solid Tumor Cell Therapy Program at UPMC Hillman Cancer Center, the program focuses on harvesting immune cells directly from a patient’s tumor, expanding and activating them ex vivo, and then reinfusing them to attack the cancer. Dr. Kammula and colleagues have been studying and using TIL therapy for patients with metastatic uveal melanoma.

The complexity of TIL therapy lies not only in its scientific underpinnings but in the multidisciplinary infrastructure required to deliver it.

Dr. Kammula’s program draws on surgical oncology, cellular processing, and specialized inpatient care to manage both the isolation of TIL, expansion of the therapeutic T-cells to doses that can cure a patient’s cancer and to deliver the cell therapy while successfully managing the associated potential treatment-related side effects. As such, it exemplifies the kind of clinical and operational sophistication needed to integrate personalized cell therapies into standard care. 

UPMC Enterprises is supporting the work of Dr. Kammula, which includes his development of a predictive diagnostic tool designed to gauge a patient’s likely response to TIL therapy for their uveal melanoma.

“TIL therapy is a clear example of where cutting-edge science meets clinical delivery,” said Dr. Billiar. “It demonstrates the importance of building not just treatments, but the systems needed to support them. We have to think holistically about how the patient is identified, how the cells are handled, and how outcomes are tracked longitudinally.” 

Cost, Coverage, and Equity Concerns of Advanced Therapeutics

Cost can often be the most significant barrier to widespread adoption of an advanced or complex therapeutic. Some treatments exceed hundreds of thousands or even millions of dollars per regimen. In many cases, payers want to wait for extensive data on safety and efficacy before they decide they will cover such therapies. 

“Academic health care centers sometimes will shoulder initial expenses to gather the data needed to demonstrate that a therapy merits broader coverage, but this approach cannot always be financially sustainable,” Dr. Billiar said.

Real-world evidence eventually determines whether a therapy can deliver enough clinical benefit to offset its price, or whether coverage restrictions will persist. 

Equity challenges can be particularly acute when life-changing medical interventions remain out of reach for patients with limited insurance or financial resources. The gap between those who can access cutting-edge care and those who cannot reflects systemic barriers that extend beyond any single health system.

Balancing Innovation with Sustainability

As an integrated delivery and finance system, UPMC is positioned to evaluate the clinical utility and economic impact of emerging therapies more holistically. This structure enables a nuanced approach to coverage decisions, factoring in potential cost offsets, such as fewer hospitalizations or long-term cures, and real-world effectiveness.

This structure means UPMC’s payer and provider divisions are more highly motivated to collaborate on the overall cost of care. “It helps us make more informed decisions on how and when to introduce these expensive, but potentially life-changing treatments,” Dr. Billiar said.

Strong data analytics and electronic health record systems also facilitate timely monitoring of performance. If real-world outcomes confirm or exceed early-phase results, an intervention may see wider coverage. If not, usage can be slowed or halted altogether. Integrated systems such as UPMC can still face challenges when an initially niche therapy suddenly expands to a broader population, driving up total costs. External payers, which may not rely on the same data or risk-sharing models, can complicate adoption if coverage decisions diverge significantly from the institution’s approach.

There are also the societal challenges posed by the high cost of advanced therapies. While continuing to lead in scientific innovation, UPMC is also focused on building a portfolio that prioritizes cost-effectiveness and long-term sustainability. This includes supporting companies like Generian, which is developing lower-cost small molecule therapies as alternatives to expensive biologics for autoimmune diseases. Additionally, there are efforts underway to advance digital tools for cost-effectiveness modeling and risk management to help bend the health care cost curve. By balancing cutting-edge science with affordability, UPMC aims to ensure that transformative treatments remain accessible and economically viable across the health care system.

Building the Future of Advanced Therapeutics, Thoughtfully

The promise of advanced therapeutics is real and it’s here right now, but so are the operational and financial challenges that are embedded in them. By investing in infrastructure, supporting novel translational science and biotechnologies, and forming strategic and targeted partnerships that can more rapidly and efficiently bring new ideas from the bench to the bedside, UPMC, UPMC Enterprises, and partners are helping to define what it looks like to responsibly develop, deliver, and monitor the use of advanced therapeutics at scale.

As innovation in the advanced therapeutics sector continues, UPMC Enterprises and the greater UPMC system are asking not only whether a treatment works, but how it can be delivered equitably and sustainably.

At the same time, phased approaches to adopting advanced therapeutics protects patients and allows institutions to gather data to advocate for better insurance coverage or refine usage guidelines. Equity, cost, and responsibility run parallel with each milestone in research and clinical care. By incorporating scientific ability with data-driven, evidence-based decision-making, UPMC’s approach to emerging therapeutics shows that a balanced strategy can leverage the potential power of advanced and complex therapeutics to achieve optimal outcomes.

“When the right science, the right people, and the right infrastructure align, we have an obligation to explore what is possible for our patients,” Dr. Billiar said.

Next Steps

• Hear UPMC Enterprises leaders in life sciences investing talk about how they are partnering with researchers to advance medicine.
• Read about efforts at UPMC Enterprises to find and support the best science and translate it into better treatments for patients. 
• Learn more about the Translational Sciences team at UPMC Enterprises. 

Note: UPMC has a financial interest in BlueSphere Bio, Avista Therapeutics, and Generian.