4 Ways Academic Medical Centers Can Capture Greater Value In The Cell & Gene Therapy Industry

By Gloria Lam and Paolo Siciliano, PA Consulting

Academic medical centers (AMCs) are unique in the way academic functions and healthcare delivery services are co-located. With strong research capabilities, access to leading scientists, clinicians, and experts in other disciplines such as bioengineers and ethicists, healthcare delivery infrastructures, and access to patients, AMCs are uniquely positioned to conduct cutting-edge research and test the efficacy of novel medicines.

Over the past 15 years, academic medical centers have played a key role in the discovery of novel therapies for unmet medical demands such as cell and gene therapies (CGTs). The first autologous CAR-T therapy, for example, was developed by researchers at the University of Pennsylvania (UPenn) and administered at the Children's Hospital of Philadelphia.

CGTs provide a novel and truly transformative solution to a number of health conditions and unmet clinical needs such as blood cancers, neurodegenerative diseases, and rare genetic disorders. While they have not yet delivered their full potential and are not yet widely adopted in clinical practice across the globe, CGTs are already changing the way we will approach drug development and disease treatment in the future.

In comparison to other therapeutic modalities, CGT represents a particularly interesting space for AMCs as the development and administration of these therapies play to several of AMCs’ key unique strengths:

• Access to patients: AMCs often have more intimate access to patients than therapy developers (such as large pharma and biotechs), and this can provide them with a considerable advantage in patient recruitment for clinical trials.
• Proximity of manufacturing and clinical delivery: For autologous therapies where blood/tissue is collected directly from the patient for processing, proximity of the manufacturing site to the hospital can greatly simplify the supply chain and potentially improve the quality of cell products due to the elimination of freeze-thaw cycles required for transportation. AMCs have the advantage of potentially co-localizing initial material collection, therapy manufacturing, and treatment all in one site.
• Agility in running clinical trials: The trial size for novel therapies for unmet medical needs is often smaller than for drug products targeting more widespread health conditions due to regulatory risk-benefit assessments. Smaller clinical trials are easier to manage and require smaller investment in early phases, providing a clear opportunity for AMCs to bring CGTs further ahead in the drug development process.
• Access to specialized facilities and capabilities: The development of CGT requires access to a broad range of skills and expertise (cell biology, bioengineering, healthcare delivery, etc.), as well as equipment and facilities (small cleanrooms, small bioreactors, clinical delivery, etc.) that are commonly available in most AMCs.

CGT products are unique and offer AMCs an opportunity to capture more of the R&D value chain than in other therapies, opening the door to additional financial and non-financial benefits for these centers, as well as increasing the speed at which these revolutionary therapies can reach proof of concept and, potentially, the market. For example, in small molecules or biologics, AMCs will have to spin out or license out straight away in preclinical research, but in CGT they can keep the R&D in-house until Phase 2 clinical trials (hence capturing more value). On top of the central role of AMCs in delivering best-in-class care for patients through offering novel treatment options and improving patient outcomes, CGTs represent a prime opportunity for AMCs to expand their research capabilities through partnerships, attract stronger network of collaborators to boost funding and innovation, and attract top-tier talent who want to work at the forefront of medical innovation.

Key Factors For Leveraging AMCs’ Unique Advantages To Develop And Deliver CGTs

Over the last few years, AMCs such as UPenn, MD Anderson, and Mayo Clinic have been very successful in spinning out/licensing CGT products and advancing patient care. After the first success with licensing a CAR-T product to Novartis in 2012, UPenn has successfully spun out 29 cell and gene therapy companies, including Cabaletta Bio, Passage Bio, Spark Therapeutics, Tmunity Therapeutics, Interius BioTherapeutics, etc. MD Anderson has partnered with Resilience to set up a joint venture, the Cell Therapy Manufacturing Center, to accelerate the development and manufacturing of both internal pipeline and external collaboration opportunities (e.g., Obsidian Therapeutics, Invectys). In January 2023, Mayo Clinic’s cell and gene therapy accelerator, Mayflower Bioventures, launched a $750 million partnership with Cellectis to create new therapies for mitochondrial diseases.

These major centers have achieved impressive results and significantly contributed to the growth of the CGT sector in different ways and through different approaches. However, their success in this space relied on a few key factors that should be areas of focus for other AMCs interested in expanding in the CGT arena:

1. Facilitating Interdisciplinary Translational Research

To successfully scale and grow CGT research and bring products to clinic, AMCs need an interdisciplinary blend of skills. For clinical research, deep scientific and technical expertise in molecular biology and genetic engineering, as well as in the specific therapeutic area of interest, is required.

AMCs should consider ways of cross-fertilizing multiple disciplines and encourage partnering and collaboration opportunities inside and outside of the institution to promote innovation. This can be done through careful consideration of the operating model and research core infrastructure to enable sharing of ideas and resources. In addition, it is important to cultivate a digital culture in academic training. Using electronic notebooks to promote digitalization of lab results and preserve an audit trail of research data, along with teaching how best to handle large amounts of data generated, are important for training the next generation of innovators in the digital age.

Another key consideration is the right mix of researchers. To bring a therapy into clinic, an institution is likely to require principal investigators (PIs) conducting translational research seasoned in clinical trial design and clinical application of products. Supporting functions, such as regulatory understanding for IND submissions, can oftentimes be a bottleneck for AMCs. Navigating FDA requirements is no trivial task and is likely to take significant effort from quality/regulatory personnel.

​​2. Enabling Process Development And Manufacturing

Developing and manufacturing experimental CGT for use in clinical trials requires the use of specialized, FDA-approved good manufacturing practice (GMP) facilities.

Establishing and running GMP facilities in academic settings require careful planning and considerations in relation to both up-front investment (financial investment, space required, location, equipment) as well as ongoing running cost (personnel, maintenance, etc.). It is important to carefully consider the user requirements for the facility and set up a facility that is fit for purpose. Depending on the product type, processes and unit operations, containment level, and the risk associated with the product, the size and configuration of the space can be very different. Consulting with experts to ensure everything is done properly and in line with regulations in the design of the facility and workflows can help prevent future operational issues.

Aditionally, while this is often overlooked, having a functioning vendor management and critical consumable supply management system (especially for reagents and consumables with long lead times) is key to reducing time and cost.

In general, for a facility to run smoothly, the key capabilities required are bioprocess engineering and quality control analytical capabilities to understand the target product profile and design space, as well as understanding GMP regulatory requirements. In addition, supporting and coordination functions for day-to-day admin activities and handling of suppliers should be considered.

3. Ensuring Sustainable And Efficient Healthcare Delivery

The processes for orchestrating trials and delivering advanced therapies (from patient enrollment to therapy administration, reimbursement, and post-administration monitoring) are highly complex. In the case of autologous therapies, implementing processes and standard operating procedures for scheduling patient procedures (sample collection, bridging therapies, infusion) and post-infusion monitoring and follow-ups is critical to improve delivery efficiency as well as reduce patient risk. Close coordination between research teams and clinicians is also extremely important to ensure sufficient capacity and successful running of trials.

In addition, significant infrastructure is needed to facilitate clinical trials. With hospitals often running at capacity treating patients with commercially available therapies, it is important to plan for additional capacity to deliver therapies in the trial stage, as well as to monitor patients post-therapy. Implementing digital systems that seamlessly integrate with existing hospital systems (both internal and external) can reduce the workload required for coordinating activities among stakeholders, especially for autologous therapies. These systems can both enable a smoother and more efficient delivery of commercially available therapies and simplify the collection of data and documentation essential for the development of new CGT during clinical trials.

From a personnel perspective, multidisciplinary teams (nursing, emergency department and intensive care unit, pharmacy, and social workers) are required for the preparation of advanced therapy delivery. In addition, the support staff who will be responsible for the communication and paperwork around reimbursement is also instrumental in the process, as, for example, negotiations with insurance companies should take place prior to the start of the clinical trials.

4. Commercialization: Building A Partnership Network Beyond Technology Transfer Transactions

AMCs usually have dedicated technology transfer, enterprise, or innovation offices to support the translation of research discoveries into commercial applications. These groups support researchers in navigating intellectual property implications of their discoveries, facilitate collaborations with industry partners, and support the developing of spinoff companies. But as mentioned by John Swartley at the Penn Center for Innovation, technology transfer is more than just supporting the transaction, it is also about making the connections and building the network.

Building an ecosystem where all partners see value in being part of the system is critical. These partners include corporations, academia, startups, facility specialists, subject matter experts, mentors, investors, government bodies, and innovation experts. Hence, the role of technology transfer, enterprise, or innovation offices in AMCs goes beyond equity and IP arrangements; it sits at the heart of the innovation engine of the center to curate resources, investments, and relationships to ensure all stakeholders remain bought in, informed, and involved beyond transactions.

Additionally, AMCs are also responsible for promoting the development of the physical and digital infrastructure to support the deeper and broader set of services. These could include physical facilities for innovators, clinicians, and investors to connect and share ideas and back-office capabilities such as legal and accounting services for spinouts.

All the above elements are necessary to enable AMCs to drive more value from the currently growing CGT industry. CGTs are realizing their potential to revolutionize the treatment of previously unmet medical needs, from breakthroughs in cancer to autoimmune and rare diseases, and AMCs are critical in pushing the boundaries in the knowledge base and clinical applications of these therapies.

AMCs are extremely well positioned to not only advance the research, development, and access of these therapies to patients but also to capture greater value in the overall CGT chain by going further in the R&D process. This will require careful planning and the implementation of the right infrastructure (physical and digital) and the right combination of skills.

That said, the development and commercialization of these therapies require significant investment in research, development, manufacturing, distribution, infrastructure, and business acumen, and it is very unlikely that one single institution will be able to build all these capabilities in-house. This, therefore, creates opportunities for deeper collaborations between academic institutions and biotechnology/ pharmaceutical companies that bring manufacturing, supply chain, and regulatory affairs capabilities in later phases of the commercialization life cycle to accelerate the development and delivery of novel therapies to serve patients in need.

About The Authors:

Gloria Lam is a cell and gene therapy expert at PA Consulting. She has a strong academic background in bioprocessing and more than seven years of international industry experience in developing growth strategies in cell and gene therapies and commercialising regenerative medicine products. Gloria holds a Doctor of Philosophy in biomedical engineering and has published articles on impact of fast-track designations, decision life cycle, supply chains, and capacity planning decisions on CGT.

Paolo Siciliano is an associate partner and life sciences expert at PA Consulting, and he leads PA Consulting’s work in CGT globally. He has several years of experience in supporting major pharma, biotech, and medtech companies to identify, develop, and leverage new technologies to solve business needs, as well as improve their innovation and product development processes. His main areas of expertise range from technology and commercial strategy to technology development, across a number of therapeutic areas. He obtained a Ph.D. in molecular biology and worked as a senior research scientist in biotech companies in the U.K.