The cell and gene therapy (C>) space is expanding rapidly. With over 2000 new gene therapy drugs in the development pipeline as of 2021, the demand for their manufacture at all scales is expected to continue to climb steadily. However, as a relatively new therapeutic area, meeting this need is becoming increasingly challenging, with manufacturing capacity, expertise, and capabilities in short supply.
In this blog, Bill Vincent, Founder & Executive Chairman at Genezen examines four key factors driving the trend toward outsourcing the development and manufacturing of C>s and offers unique insight into how this could change in the future.
Meeting demand in the growing C> space
In the last two decades, significant advancements in genetic engineering, molecular techniques, and technologies have revolutionized the C> space. Previously limited to treating rare and orphan diseases caused by genetic abnormalities, progressive innovations have helped to broaden C> applications. Drugs in the development pipeline now aim to treat indications ranging from cardiovascular diseases to metabolic conditions and respiratory diseases while offering the potential to bring long-term benefits1.
With growing numbers of biopharma companies beginning to realize the potential of these drugs and over 2000 gene therapies in the development pipeline1, growth of the C> space is unlikely to slow. In fact, the global outlook of the gene therapy market is predicted to rise from a value of USD $4.99 billion to USD $36.92 billion between 2021 and 2027, at a compound annual growth rate (CAGR) of 39.62%2.
Having the necessary manufacturing capacity and capabilities will be critical to meet this growing demand for C>s. Although biopharma companies continue to invest in infrastructure to ensure these needs are supported, outsourcing to specialist contract development and manufacturing organizations (CDMOs) is becoming an increasingly attractive prospect.
The key drivers in seeking manufacturing support
In a recent report, 44% of biopharma companies were found to outsource most or all of their C> manufacturing activities3. It is not surprising that so many in the biopharma industry are looking to CDMOs to support their C> production efforts; there are many factors driving both small and large organizations to rely on development and manufacturing partners.
1. A surge in startups
The past five years have seen a significant increase in venture capital (VC) funding and investment into startup biopharma companies. By 2016, VC companies were found to have invested in 2,200 biotech start-ups worldwide, growing to 3,100 in 20214. Many of the C>s currently in the pipeline have consequently been developed by startup companies.
Inherently, these new startups will often lack the existing capacity needed for manufacturing and, being early on in their journey to market, they generally lack the necessary experience to handle the challenges that might come their way. Requiring robust processes, quality programs, and a solid understanding of GMP regulatory requirements, these startups also benefit from the support of experienced CDMO partners with specialized capabilities and facilities.
2. Weighing up the costs
Even for larger biopharma companies, finding appropriate manufacturing facilities can be as challenging as it is for startups. These organizations must weigh up the capital costs of construction and the expense of specialized equipment versus contracting out to those with facilities and capabilities already in place.
However, the capital investment required to build a manufacturing facility is often substantial. A small pilot plant with 100 L cell culture capability could cost up to $10 million, and a 1,000 L scale plant could set these companies back up to $40 million. Larger still, building a commercial plant offering a capacity of 10,000 L capacity can require up to $200 million5-7. Although a hybrid option could offer a suitable alternative for small companies with limited capital investment, the cons of building facilities often outweigh the pros until significant funding has been attained.
3. Navigating commercialization roadblocks
Following the onset of the pandemic, demand for drug manufacturing capacity rose steeply. CDMOs were in high demand and had to offer flexibility to balance the needs of vaccine projects with those of other clients. Despite best efforts, delays were often seen due to capacity and supply limitations, putting pressure on both biopharma and CDMOs to invest in facility expansions.
For biopharma companies, building new facilities or retrofitting an existing one can cause further delays in the journey to commercialization. High demand for design, construction, and consulting services has led to a shortage in access to those with the necessary skills to put expansion plans into place.
Fortunately, many CDMOs, including Genezen, have taken on the challenge of expanding to offer additional process development, analytical development, and quality control labs as well as cell banking and manufacturing suites to relieve capacity pressure. As projects progress through the pipeline, these facilities will further help alleviate bottlenecks in production scale-up.
4. Lack of specialist expertise
A prevalent challenge for drug developers in the C> space is the difficulty finding those with the necessary expertise to deliver projects to critical milestones – particularly in viral vector development and manufacturing. Trends in lentiviral and gammaretroviral applications for C>s and the fact that viral vectors are a relatively new technology mean that there are few development and manufacturing specialists in the area.
Lack of skilled labor can delay the progression of drug candidates to commercial production, particularly for small biopharma companies and startups with smaller internal teams. Limited access to specialist skills is considered to be a top driver toward the decision to work with a CDMO3, allowing C> drug developers to forgo the difficulties in hiring and training personnel.
Looking to the future
As the C> space matures, sponsor companies will have to continue carefully consider whether to invest in internal capacity or to rely on the support of external partners. Efficiency, cost-effectiveness, and expertise will all come into play when determining the best path of least resistance.
Offering specialist expertise, experience, and capacity, CDMOs are set to remain key to delivering new C>s to market, with the average number of C> CDMOs that biopharma companies rely upon expected to increase in the next five years3. Many of these CDMOs are once again proactively preparing for the future needs of their clients, designing new manufacturing spaces with flexibility in mind to keep up with innovations and regulatory changes.
To learn more about how Genezen’s own expansion and overall master plan for a 75,000-square-foot lentiviral and retroviral process development and cGMP vector production facility could support your next project, explore our facilities page or contact us today.
- American Society of Cell + Gene Therapy. Gene, Cell, & RNA Therapy Landscape. Q3 2022 Quarterly Data Report.
- G. Pugh, “Cell Culture Manufacturing COG: A Lifecycle Approach,” ISPE conference, Oct. 12, 2000.
- T. Hartnett, “Computer Models and their use within a Supply Chain Organization,” IBC Production and Economics conference, Sept. 29 – Oct. 2, 2003, San Diego, CA.
- E. Miller, “Build New, Retrofit, or Outsource?” IBC Retrofitting & Building Facilities for Biopharmaceutical Production conference, Nov. 12-13, 2001, San Diego, CA.