The Emergence Of New Gene Therapy Hubs
By Timothy Miller, Ph.D.
This article originally appeared in Life Science Leader magazine.
Within the broader field of biotechnology, the focus on gene therapy has expanded rapidly in recent years. The primary reason is that many promising development programs are reaching later stages and are headed for the finish line. Recently, the FDA’s advisory panel unanimously backed a new gene therapy intended to treat a rare eye disease, potentially leading to the first FDA-approved gene therapy in the U.S. Our progress in this sector is expanding and may lead to gene therapies that can deliver curative treatments for many significant areas of unmet need in healthcare in the near future. As these development programs advance, a growing need is presented for gene therapy research and manufacturing centers.
More than 30 years ago, at the dawn of the biotechnology era, the first generation of companies set up shop in areas that offered several key attributes, including a science-educated workforce, proximity to leading research centers, access to capital, space to accommodate often custom-designed technologies, and tax breaks and other incentives. These factors gave birth to the established hubs in Cambridge and Silicon Valley and also have provided support for smaller hubs in locations including Ann Arbor, MI; Philadelphia; and Denver. With gene therapy, we are at the dawn of another era of potentially rapid growth in the sector. And companies planning for their futures will once again consider a range of factors in identifying the optimal locations for gene therapy research and production centers.
THE NEW BIOTECH HUBS
Since the founding of the first biotechnology companies, a few things have changed that will affect where gene therapy producers may choose to set up operations. Many of the established hubs are no longer as eager to attract new companies, and so tax breaks and other incentives may be less generous. There is also intense competition for employees and access to research centers in those areas. In some cases, housing costs and other quality-of-life factors present challenges in attracting employees. But one of the most important factors is that there are now many options for locations where access to science-educated employees, established research centers, capital, and state-of-the-art technology is possible. As a result, leaders in gene therapy have considerable flexibility in choosing a location for future facilities.
One well-positioned option is Chicago, which is now among the top locations in the U.S. to receive funding from the NIH as well as venture capital investment. Chicago’s thriving Illinois Medical District (IMD) now includes 560 acres of medical and healthcare-related facilities, labs, and several major hospitals including Rush University Medical Center. Local institutions such as Northwestern University, The University of Chicago, and the University of Illinois at Chicago also offer potential support from academic leaders involved in research.
Also in the Midwest, Indianapolis is a prospective biotechnology hub with many advantages. It is the headquarters for Lilly and home to the growing Indiana Biosciences Research Institute, which currently includes 18,000 square feet of laboratory and office space and is situated between four major hospital systems. There are also many public and private universities that feed into the local biotechnology industry, including Indiana University and Purdue University. With these resources nearby, many companies will be able to find the talented workers and innovators they need to facilitate development of advanced gene therapies.
The Research Triangle Park in North Carolina is another quickly growing area for biotechnology companies. It is already one of the largest research parks in the world, with more than 250 businesses and a network of medical researchers from centers including the National Institute of Environmental Health Sciences and the North Carolina Biotechnology Center. These resources are well-positioned to meet the needs of many gene therapy companies.
On the West Coast, Los Angeles is building its reputation in the life sciences sector. While the area does not have as big a foothold in biotechnology yet, it is the home of notable research centers including UCLA, the California Institute of Technology, and the Los Angeles Biomedical Research Institute. There, employers can tap into a highly skilled pool of life sciences graduates and medical researchers.
Cleveland, the site for Abeona’s new gene therapy manufacturing center, is now a leading area for investment in biotechnology. The area attracted more than $2.17 billion in funding in 2016 from sources including venture capital, angel investment, federal research grants, and state initiatives such as the Ohio Third Frontier. Companies received private investment worth $373 million in 2016 and estimates indicate at least $155 million has been invested so far in 2017. It is also the home of leading healthcare businesses and research centers including BioEnterprise, Cleveland Clinic, HealthTech Corridor, and Midtown Cleveland. The affiliation with Nationwide Children’s Hospital, which is in nearby Columbus, also made Cleveland a highly attractive option for our company.
CONSIDERATIONS IN MANUFACTURING
One important factor that growing companies may not consider early on when choosing a location is the need for future expansion of their manufacturing operations. Companies focused on developing gene therapies especially need to plan for manufacturing needs at every stage of clinical development and be prepared to meet demand for products at commercial stage. They can face significant problems if they cannot expand their footprint as production needs escalate. Companies must consider future needs related to scale-out, use of off-the-shelf versus custom technology, and risks associated with variability as production needs escalate. Investing in facilities that can handle these complex manufacturing needs is critical.
It is also important to monitor the local workforce and educational resources to make sure they are continually positioned to support future staffing needs. Research centers and educational facilities must be able to produce trained workers with the advanced skills and experience gene therapy companies require. At Abeona Therapeutics, we are developing next-generation, adeno-associated virus (AAV) gene therapies. When choosing a location for our company, we had to confirm access to a pool of employees and suppliers who understand the complex manufacturing associated with our AAV vector platform. These needs will differ based on the specific type of gene therapy being produced, and it is unlikely that any company will find a team of experts in their unique manufacturing process available at every stage of their growth. In addition, many gene therapies are targeting treatments for rare diseases, which also can require specialized training. Companies must do their due diligence to assess whether a region can accommodate their future needs in both skilled employees and advanced technologies.
Many key regions are both underappreciated and undervalued, which can be an advantage to emerging gene therapy companies. They present compelling combinations of available space, affiliation with leading academic research centers, local incentives, good quality of life, and other advantages. Companies should consider all of these factors and many others — and assess each option based on multiple considerations. But the bottom line is that companies should not be afraid to be a big fish in a small and relatively unknown pond. There can be less competition, reduced operating costs, and a pool of eager untapped talent compared to the bigger hubs. Companies also can find locations that offer great flexibility for future expansion. For example, in Cleveland, we have found and developed a talented melting pot of academia, biotechnology, patient advocacy, and other expertise necessary for commercial success — a community that we are proud to call our own.
By Timothy Miller, Ph.D. is president and CEO of Abeona Therapeutics and has 16 years of scientific research, product development, regulatory, and clinical operations expertise.