The rapid expansion and advancement of the cell and gene therapy pipeline is forcing companies to rethink their early stage priorities. With nearly 1,000 companies competing for finite production capacity, forward-thinking leaders are recognizing the value of establishing manufacturing strategies far earlier than is typical for other modalities and engaging with external development and manufacturing partners to secure long-term supply capacity.1
At the start of 2015, sponsors were running 486 clinical trials of advanced therapies.2 By the end of 2019, the figure had more than doubled to 1,066. The trend was driven by an increase in phase 2 and 3 clinical trials, which accounted for 685 of all advanced therapy studies tallied in the last count. The rapid expansion is set to continue, with the FDA expected to receive more than 200 cell and gene therapy INDs a year starting in 2020 and approve up to 20 products a year from 2025 onward.3
The figures tell the story of a sector that has quickly gone from making therapies in hospital settings to treat small numbers of patients, to running industrialized manufacturing processes that ship drugs to patients at commercial scales.
Why Manufacturing Decisions Cannot be Deferred
Some of the 200-plus cell and gene therapies that enter the clinic every year are likely to advance quickly into late-phase clinical trials and commercial use. As medicines that almost exclusively target major unmet medical needs, many cell and gene therapies have regulatory designations that speed their progress into large trials, further increasing the number of doses the industry needs to make.
With many more patients receiving cell and gene therapies than ever before, some CDMOs and Big Pharma companies are adding capacity. Even so, the pace of the pipeline expansion has outstripped the growth of capacity, leading William Blair analyst John Kreger to note that companies that want to outsource production face the prospect of joining “elongating waiting lists.”
In this environment, companies cannot defer decisions about manufacturing. Companies developing monoclonal antibodies can get away with focusing on other matters early on, safe in the knowledge that standardized processes and established infrastructure will simplify scale-up when they turn their attention to manufacturing. Cell and gene therapy companies lack that luxury.
The absence of standardized processes for manufacturing cell and gene therapies, and in some cases the need to make treatments personalized to individual patients, means even in a world of abundant capacity startups in the field would need to consider production early to avoid bottlenecks.
Startups that fail to recognize the need to prioritize manufacturing early will struggle to move quickly through development. Growing recognition of the problem means companies may even be unable to secure the funding they need to reach the bottleneck. After seeing startups struggle to source batches for trials, Dmitry Kuzmin, managing partner at advanced therapy venture fund 4BIO, began pushing for his portfolio companies to have “a coherent manufacturing strategy from very early on.”4
How Partnering Provides a Competitive Advantage
Early stage, venture-backed biotechs are unlikely to have the pipeline to justify establishing in-house capacity. Even if they did, it is likely that, in such a fast-changing, non-standardized field, they would be better served by outsourcing to a partner that could bring experience and expertise gained across a large number of programs to bear on their manufacturing processes.
Those considerations are leading cell and gene therapy companies to identify CDMOs as the best way to set up the manufacturing plans they need to avoid bottlenecks later in development and, in the case of venture-backed startups, show investors they are equipped to move quickly to market.
CDMOs have the process development and manufacturing expertise to truncate timelines, helping companies get cell and gene therapies to patients sooner, and can free partners from the need to add internal capabilities in areas ranging from raw material sourcing through to clinical trial supply.
The imbalance between supply and demand means companies may struggle to access those benefits at short notice. Yet, by planning ahead and engaging an outsourcing partner early, companies can secure everything they need to take cell and gene therapies through late-phase development and on to the market. The partner will have dedicated space ready for when the client needs it and then increase capacity in line with their requirements as they start to treat more and more patients.
Companies that avail themselves of the support CDMOs provide early in development stand to gain an advantage over their peers. With a manufacturing strategy that stretches to commercialization in place, companies can focus on R&D confident in the knowledge that industry-leading experts will handle the task of getting drugs to the patients who need them, when they need them.
References
- ARM Annual Report & Sector Year in Review: 2019. https://alliancerm.org/publication/2019-annual-report/.
- Q1 2015 Alliance for Regenerative Medicine Quarterly Data Report. https://alliancerm.org/wp-content/uploads/2018/04/ARM_Q12015_Data_Report_Web_Version.pdf.
- Office of the Commissioner. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. U.S. Food and Drug Administration https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics (2019).
- Taylor, N. P. 4BIO raises VC fund to take cell and gene therapies mainstream. FierceBiotech https://www.fiercebiotech.com/biotech/4bio-raises-vc-fund-to-take-cell-and-gene-therapies-mainstream (2019).