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By Richard Cassidy, Head of Operations & Commercialization, Astellas Institute for Regenerative Medicine

The US FDA expects that by 2025 it will approve 20-25 regenerative medicine products annually. This new class of therapeutics — including cell therapies, gene therapies and stem-cell based therapies — is on a steep growth trajectory, yet many challenges remain to bring these potential innovations to patients.

The path from lab to clinic to patient, for each new medicine in this novel class of therapeutics, presents a unique set of drug development and commercialization challenges that are different from typical pharmaceuticals and biologics. To realize a meaningful difference in care, all stakeholders in the healthcare ecosystem must understand how they can contribute to bringing these potentially transformative therapies forward.

Collaboration is key

Innovators in this space must build, grow and partner to establish the specialized capabilities required to tackle the unique challenges of regenerative medicine. Already, there has been a steady flow of partnering deals and collaborations in the cell and gene therapy space and, as the field continues to grow, so too will the pace of deal-making.

From early research to clinical development to manufacturing and commercialization, the expertise and capabilities needed often reside in different types of companies. Smaller biotech companies may have unique technologies and scientific expertise, but limited resources. Larger pharmaceutical companies know how to develop and deliver drugs but may not have early scientific expertise in the field. Thus, companies in the cell therapy space should be focused on evaluating their own strengths and weaknesses to determine opportunities to source needed capabilities through complementary collaborations.

Both ends of the spectrum

Capabilities in drug development can be broadly divided into two segments:

  • Creating value through innovative technology and R&D;
  • Delivering value through product commercialization.

Creating value: The spark of innovation

Novel regenerative medicine approaches bring new scientific and R&D complexities that are different from developing drugs in established modalities, such as small molecules or antibodies. For gene therapies, viral vectors are expensive and time-consuming to produce and characterize. For certain cell therapies, there can be challenges with production of cell lines, which can limit the quality and quantity of raw materials.

In today’s landscape, the pioneering element in novel cell and gene therapies is often the domain of start-ups and young biotech companies that combine scientific and entrepreneurial capabilities to rapidly translate academic technologies and ideas into innovative cell therapies that can move toward becoming a clinical reality. Yet, even after those early discoveries, many steps remain to realizing the potential of these therapies.

Delivering value: The path to the patient

Even after achieving clinical proof of concept, there are still myriad additional hurdles ahead to produce a true medicine, including:

  • Manufacturing: Achieving consistent and controlled production is one of the most unique and challenging components of creating a cell therapy. By their very nature as living cells, cell therapies are complex and variable, and can present challenges in product testing and characterization required for a safe and robust manufacturing process that follows Good Manufacturing Practice (GMP) principles.
  • Supply Chain: Therapies from living cells, being highly sensitive to time and temperature, can create supply chain challenges and, in many cases, require new technology solutions and monitoring to ensure end-to-end traceability. New models for distribution networks are emerging and for this class of medicines to become mainstream, the healthcare ecosystem must adapt.
  • Pricing and Access: The prospect of drugs that enable the body to heal itself with a single treatment requires an entirely new model for pricing and access. The first wave of approved cell therapies is paving the way for important discussions about a treatment’s value — including pricing and reimbursement models — so that patients can access these potentially curative medicines.

A new field of vision

Cell and gene therapies represent far-reaching opportunities for patients and innovators alike. To be successful, each stakeholder in the ecosystem needs to look to the future and consider how they can contribute to creating and delivering on the promise of regenerative medicine for patients. In the end, the path forward will likely include partners and collaborations.

Richard Cassidy is the Head of Operations & Commercialization at the Astellas Institute for Regenerative Medicine located in Marlborough, Massachusetts.

Astellas contributed this perspective as part of our commitment to turn innovative science into value for patients in many areas, including our work in regenerative medicine. On the forefront of healthcare change, Astellas continuously collaborates to advance ground-breaking science in new technologies across multiple areas of biology and disease. Learn more at