Regenerative Medicine: Engineering Its Continued Success

Like many advanced technologies, the field of regenerative medicine has gone from boom to nearly bust to boom again in the span of just 30 years. Today, there are over 55 regenerative medicine products on the market focused on diverse therapeutic areas, including repair of skin/soft tissue, wound care, cardiology, oncology, and diabetes.ⁱ Thirty years in, regenerative medicine has truly “come of age,” the result of a tenacious pursuit to translate groundbreaking research into therapeutic products and overcome initial setbacks that almost derailed this critical new medical approach.

Yet while the past decade’s focus on scientific advances and business fundamentals has propelled regenerative medicine forward, I believe this is just the start. By reflecting on the successes and lessons learned over the past three decades, we can begin to chart a roadmap for the future that will help to ensure that regenerative medicine continues to deliver important new treatments for patients, while creating sustainable value for shareholders.

The First Bubble

From its origins in the mid-1980s, regenerative medicine was greeted with the kind of extreme excitement that has accompanied other potential breakthroughs, including monoclonal antibodies and RNA interference. By the year 2000, more than a decade after the first companies were formed, regenerative medicine companies were valued at over $2.6 billionⁱⁱ, TIME named tissue engineering one of the hottest jobs for the 21st century, and Barron’s predicted it would become a $100 billion industry. A few years later, the bubble had burst, and company valuations plummeted to a tenth of their year 2000-high.ⁱⁱ

Several factors contributed to these setbacks. First, like many new medical advances, expectations far exceeded reality. Investors and the media saw incredible promise in early research, and unrealistic timelines were set for when a product could be on the market. Second, the initial regenerative medicine products to reach the market had limited commercial success, as the few companies in the space had not yet understood all that was required to achieve both clinical and commercial success. From a scientific perspective, the field was poised to deliver, but it had not yet developed the regulatory, business, and commercial expertise required for long-term success.

A Decade of Growth

In the wake of these setbacks, there came a clear understanding of what was needed to propel regenerative medicine forward and strike the appropriate balance between promise and reality. When I joined Organogenesis in 2003, the company was emerging from bankruptcy and a dissolved commercial partnership with big pharma. In the decade since, I have experienced firsthand the rebirth of our company, and on a larger scale, of the regenerative medicine field itself. Our path over the past decade has taught us several lessons about what it will take to succeed in this space going forward. In the years ahead, I believe the regenerative medicine field will continue to grow and adapt in the following ways:

• First, regenerative medicine companies will each individually focus on a small number of high-priority areas and avoid the temptation of pursuing too many opportunities at once. This will be especially critical since the FDA reviews most regenerative medicine products through the Center for Biologics Evaluation and Research (CBER). As a regenerative medicine company, we learned firsthand that it requires substantial investments of time and capital. Ultimately, this process is healthy for the industry and should place those that go through it on a more stable and sustainable path.
• Second, companies will continue to de-risk their clinical development programs by utilizing more effective preclinical models to select and move forward with viable potential product embodiments. With continued interest in regenerative medicine, the clinical development environment has become increasingly competitive, giving rise to more sophistication of the R&D enterprise. Going forward, it is more important than ever to use scientific, risk-based approaches (i.e., “Quality-by-Design”) during process and product development. This systematic practice not only increases the organization’s knowledge space, it also empowers the company to better calculate decisions designed to avoid possible bottlenecks or “surprises” during late-stage development.
• Third, our field will continue to grow by fostering the commercial expertise needed to build value for regenerative medicine products. Over the past decade, an acute recognition of the many unique aspects required for commercial success in regenerative medicine has led pioneering companies to invest in building their own sales and marketing organizations, rather than outlicensing to the major pharma/device players that are becoming more keenly interested in entering the field due to the value potential for this innovative therapeutic approach.
• Finally, regenerative medicine companies will more fully embrace the reality that scaling up a living, cell-based product requires unique skills in R&D, quality, process development, process engineering and production. As the field evolves, it has become clear that the major barrier to entry is less about intellectual property than about the complexity and cost of operations. In line with this understanding, we are taking the lead in building an automated living cell manufacturing plant, an initiative that will strengthen capabilities in the field.

Regenerative Medicine’s Impact: A Look Ahead

As our healthcare system faces an aging population and an increasing prevalence of chronic diseases, regenerative medicine will continue to play an increasingly important role in patient care, particularly where we offer therapies that deliver benefits that elude traditional drugs, devices, and surgery. Our unique ability to harness the body’s own healing processes in a safe and targeted manner means that we will play an increasingly important role in the management of difficult-to-treat diseases. More than 17,000 patients are expected to be involved in regenerative medicine-related clinical trials,ⁱ demonstrating that the pipeline of therapies on the horizon continues to expand.

Now that the first living allogeneic cell-based therapy (GINTUIT) has been approved through the FDA via CBER, we have achieved a major milestone: the first step in establishing consistent approval pathways. This clarity is critical to ensuring that a high level of scientific and clinical integrity is applied to all regenerative medicine products, which in turn will provide a clear differentiation from the growing number of unproven products that are flooding the market today with dubious claims that are unsubstantiated by rigorous clinical research. This is an important step in the continued maturation of the field, and a key value-driver for industry as we work to demonstrate the value of our therapies for physicians, patients, and payors alike.