A Summary of Cell & Gene Therapies Market Outlook Report
By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1
As seen in Life Science Leader magazine
Even if your company isn’t developing and manufacturing cell and gene therapies (CGTs), you should be keenly aware of their almost limitless future therapeutic implications.
As such, I want to bring to your attention an important research report, Cell & Gene Therapies Market Outlook, released in Q3 2021 by Industry Standard Research (ISR). The report is based on data gathered from 101 respondents from North America, Europe, and Asia via a 15-minute web-based quantitative survey. This is the first edition of the report, and it was designed to provide support and direction for innovator companies with cell and/or gene therapies in their pipelines or portfolios as well as to CDMOs that are looking to win their manufacturing business.
The report provides an overview of the current market dynamics in the CGT space, as all survey respondents are outsourcing decision makers for CGTs at sponsor organizations. It is broken down into five parts: market dynamics, outsourcing preferences and practices, service provider selection, cell and gene outsourcing challenges, and the study data. The latter includes the full data set in chart form. Responses predict what the marketplace will look like in 2026. Here’s a summary of ISR’s findings.
MARKET DYNAMICS
On average, respondents with a cell therapy offering have roughly five cell therapies in their pipelines while those with a gene therapy offering have approximately four therapies in theirs. The oncologic therapeutic area claims more than half (58%) of the respondents. Approximately one-quarter of respondents have CGTs focused on the hematology (28%), infectious disease and vaccines (27%), immunology (27%), and neurology (26%) therapeutic areas.
Annual Outsourcing Spend On Cell & Gene Therapies By Company Size
Most respondents were from large ($5B+) companies (58%) and are spending more than $50 million annually on outsourcing CGTs. Conversely, 43% of respondents from non-large (<$5B) companies are spending less than $10 million, and 39% of respondents from non-large companies are spending $10 million to $50 million on outsourced CGTs.
OUTSOURCING PREFERENCES AND PRACTICES
According to Cell & Gene (the website I manage), it is estimated there are more than 1,000 clinical trials in cell and gene therapy ongoing globally. Many new therapeutic approaches require the production of viral vectors, yet viral vector manufacturing is a cumbersome process. ISR’s report corroborates this, as it states that viral vectors are the most common CDMO need among respondents, with 62% of respondents stating they will outsource viral vector manufacturing to CDMOs over the next 18 months. That percentage jumps to 68% in five years. Plasmid DNA follows distantly with 46% of respondents indicating they will outsource this capability now and 50% in five years. One-third of respondents mentioned they will outsource allogenic cell therapy manufacturing activities in the next 18 months, a metric that is anticipated to increase by 9 percentage points over the next five years. Thirty-seven percent of respondents will outsource autologous cell therapy manufacturing in the coming 18 months, with that percentage expected to increase to 42% in five years.
Viral Vector Outsourcing Needs
Of those respondents who will outsource viral vector manufacturing activities over the next 18 months, half will use adenovirus technology. About one-third of respondents use the lentivirus (38%), and one-third use adeno-associated virus (AAV) (35%) for viral vector manufacturing. One-quarter use oncolytic virus for their viral vector manufacturing needs.
Allogenic Cell Therapy Outsourcing Needs
According to ISR, most respondents with allogenic cell therapies in their pipeline will require cell-based immunotherapy (84%) technologies and services from CDMOs. Induced pluripotent stem cells (iPSC) manufacturing (29%) and exosomes (26%) follow with just over one-quarter of respondents with allogenic cell therapies using these technologies and services.
Autologous Cell Therapy Outsourcing Needs
ISR states that a greater proportion of respondents with autologous cell therapies in their pipelines will use viral modification (84%) as opposed to nonviral modification (49%).
Outsourced Cell & Gene Activities and Services
Currently, clinical supply logistics (50%) and process development/scalable manufacturing processes (49%) are the most-outsourced activities among respondents. The proportion of respondents outsourcing these activities is projected to remain steady five years from now. Fill/finish (44%) and primary packaging (40%) follow in terms of current CDMO needs among respondents. The activities where respondents anticipate the most growth over the next five years are secondary supplier to support risk management, which is predicted to have a 15 percentage-point increase by 2026, and primary supplier, with an increase of 11 percentage points.
Manufacturing Findings: A Shocking Truth
Cell and gene therapies demand a manufacturing approach that is more decentralized. They also introduce significant new logistical challenges with key parts of the process occurring in hospitals or clinics, within cold chain transit, or in a manufacturing facility. Manufacturing equipment, in turn, needs to be ultra-mobile and sized to sit at the patient’s bedside.
Respondents stated that the most common stage of development among respondents to identify a CDMO for commercial manufacturing needs for CGTs is during the preclinical stage (27%). Phase 2 follows closely with 23% of respondents starting to look during this phase.
ISR found that currently more than two-fifths of respondents outsource 91-100% of their company’s cell and/or gene therapy manufacturing activities. And now for the shocking truth: This figure is predicted to decrease by half (yes, half) over the next five years, dropping to one-fifth of respondents outsourcing between 91% and 100% of cell and gene manufacturing as biopharma companies increase or establish in-house manufacturing capacity. We can dedicate a whole other article to the “why” behind CGT companies’ in-house manufacturing considerations.
SERVICE PROVIDER SELECTION
Unsurprisingly, when it comes to choosing a supplier, executive management holds the most influence over the decision (21%), followed by manufacturing/production (15%). Most respondents (86%) expressed moderate or greater importance in using the same CDMO for both development-stage and commercial manufacturing. And technical capabilities and technical service take the top slot as the leading satisfaction drivers when working with CDMOs for outsourced CGTs.
Then there are the types of service providers used by CGT companies. Cold chain logistics leads (55%) followed by midsized, full-service CDMOs (50%) and large, global CDMOs (47%), and so on. Cold chain logistics taking top score is no surprise, as the cold chain for a cell therapy product must be capable of maintaining a living product in a viable state throughout storage and distribution, all the way to administration to the patient — a uniquely challenging but non-negotiable criteria.
CELL & GENE OUTSOURCING CHALLENGES
In line with the top listed area on which to focus technology and infrastructure investments, and the most outsourced activity currently, top challenges when outsourcing cell and gene manufacturing activities are scalability (54%), cost (47%), and reproducibility (46%). Analytical technologies and logistics/transport are also mentioned as challenges among one-third of respondents.
As part of the survey, ISR asked its respondents, “In your opinion, what is the most difficult part of outsourcing cell and gene therapy manufacturing activities?” and their written responses were telling. One respondent stated, “Getting suite space. Very long lead time for reserving manufacturing capacity.” According to Cell & Gene, while the CDMO market is growing, so too is the rapid expansion of the CGT pipeline, which creates constraints on access to contract capacity. There just isn’t enough manufacturing capacity for every company that wants to take drug candidates to the clinic. As such, companies might face a 12- to 18-month wait before a CDMO can begin work on their project. Another respondent lamented that the living and unique nature of every CGT adds to the complexity of outsourcing. Another stated that finding a CDMO that has a granular understanding of how to upscale in a compliant and rapid fashion is a challenge.
Deciding which CDMO to take them from bench to bedside and, ultimately, commercial supply is one of the most critical decisions CGT companies face. Outsourcing preferences, practices, and challenges, as well as selecting the right solutions provider will always be top of mind for CGT decision makers whether it’s today or five years from now as evidenced by the data in ISR’s Cell & Gene Therapies Market Outlook report.