Building a regenerative medicine industry: Lessons from North Carolina

The field of regenerative medicine is advancing rapidly, offering transformative solutions for a wide range of health conditions. However, while scientific breakthroughs in regenerative medicines like cell and gene therapies continue, the ability to transition these innovations from the research phase into commercially viable treatments remains a more significant challenge.

North Carolina’s regenerative medicine ecosystem has developed a strategic approach to addressing these challenges. Organisations and accelerators worldwide can learn from this model to enhance their own therapeutic development efforts, in the field of regenerative medicine or in other similar emerging areas of healthcare where the causes of product failure have been established.

A strategic focus on the development phase

Regenerative medicine faces an exceptionally high attrition rate, with over 90% of cell and gene therapy candidates failing before reaching commercialisation. Unlike traditional pharmaceuticals, where failure is often due to safety or efficacy concerns, the primary challenge in regenerative medicine is in the development phase – specifically, the ability to consistently produce quality therapies at a cost that is acceptable to the market.

Recognising this gap, the launch of the Piedmont Triad Regenerative Medicine Engine (PTRME) was supported by funding from the National Science Foundation last year, and since that launch has prioritised manufacturing, supply chain capability, and quality control as key areas for investment. This focus ensures that scientists and companies working on promising regenerative therapies can overcome barriers that often prevent commercial viability, most commonly hung up in the product development stage.

A model for other markets and therapeutic areas

The model employed by North Carolina’s regenerative medicine ecosystem can be adapted by other accelerators and organisations looking to support nascent therapeutic programmes looking to focus on turning innovative ideas into real, life-saving applications. Key components of this approach include:

1. Prioritising development over research – While research is essential, commercial success depends on the ability to transition discoveries into viable products. Establishing dedicated funding and resources for the middle step, the development phase, is highly needed in the biotech marketplace today.
2. Addressing manufacturing and supply chain challenges – Many regenerative medicine failures result from the inability to manufacture therapies at scale. Accelerators should work with industry partners to develop solutions that streamline production and reduce costs in order to create products at price points that can then be marketed through commercialisation.
3. Leveraging public-private partnerships – The success of the PTRME is built on collaboration between academic institutions, industry partners, and federal agencies. Similar initiatives should seek diverse funding sources, including government grants, venture capital, and private equity, in order to establish a long-term ecosystem for the advancement of new therapeutic technologies.

The role of policy and regulatory support

Regenerative medicine innovation is influenced by regulatory frameworks that vary by region. Europe and Japan, for example, have approved significantly more regenerative medicine therapies than the United States, partly due to streamlining of advanced therapy approvals (e.g., the Advanced Therapy Medicinal Products (ATMP) pathway in EU). Other markets should examine regulatory policies that have successfully accelerated approvals while maintaining safety and efficacy standards, and be willing to work on the advocacy and policy side required to advance their ecosystems.

The US Food and Drug Administration (FDA) is making progress in this area, with recent indications that cell and gene therapies may become more competitive even than traditional small and large molecule therapies. As the regulatory landscape evolves, industry stakeholders must engage with policymakers at all governmental levels to advocate for frameworks that support responsible innovation.

North Carolina’s unique advantages

North Carolina’s success in regenerative medicine is due in part to its well-regarded life sciences industry, but equally also due to its strength in manufacturing. The state’s history in manufacturing, particularly in the central Piedmont Triad region, provides the foundation for scaling regenerative medicine production. Companies and accelerators in other regions should assess these kinds of complementary local strengths when determining their ecosystem building strategies.

With a workforce well-trained for manufacturing, the regenerative medicine innovations will undoubtedly be able to move through development within the same market as where the research is happening. Thanks to Dr Anthony Atala’s decision to move from Harvard University to Wake Forest University more than 20 years ago to build on his remarkable momentum in regenerative medicine research, the industry now is well-aligned to provide innovative new jobs to manufacturing talent within the state.

Looking ahead

The regenerative medicine industry is reaching a critical point where development and commercialisation efforts must keep pace with scientific innovation. North Carolina’s approach demonstrates that targeted investments, industry collaboration, and a focus on manufacturing and supply chain efficiency can drive success. By adopting similar strategies, accelerators and organizations around the world can enhance their ability to bring life-changing therapies to market.

About the author

Tim Bertram, PhD, is the new chief ecosystem officer of the Wake Forest Institute of Regenerative Medicine and serves as CEO of the Piedmont Triad Regenerative Medicine Engine. With an illustrious career marked by innovation and leadership, Bertram has decades of transformative industry experience from his tenure at ProKidney, a chronic kidney disease therapy company he founded focused on preserving kidney function and controlling kidney failure. Prior to founding ProKidney, Bertram held pivotal roles in various capacities, with a deep-rooted commitment to advancing science and technology, with companies such as inRegen, Tengion, Inc., and Pfizer, helping to bring eight new medicines into the marketplace. Bertram graduated from Iowa State University with a degree in Biology before gaining his Doctor of Veterinary Medicine and PhD in Cellular Pathology from the same institution. He completed Post-Doctoral Research in Cytokine Biology with the NIH and is board certified with the American College of Veterinary Pathology (DACVP).