A Summary Of Cell & Gene Therapies Manufacturing Outlook Report: 2023 Edition
By Erin Harris, Editor-In-Chief, Cell & Gene
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Last year, I wrote a summary of Cell & Gene Therapies Market Outlook, a research report released in Q3 2021 by Industry Standard Research (ISR). The original report was ISR’s first edition, 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 were looking to win their manufacturing business. ISR just released Cell & Gene Therapies Manufacturing Outlook (2nd edition), and just like its predecessor, it’s chock full of valuable data.
Visit ISR for the full-length report, and in the meantime, here is a summary of their findings.
After having cleared a multi-step screening process, 100 respondents completed a 15-minute quantitative online survey, and data were collected in Q1 2023. Here is a snapshot of the respondents’ demographics; note these stats include some but not all the demographic information contained in the report. Biopharmaceutical companies (R&D $1B or more) represented 44% of respondents, and biopharmas with R&D less than $100M represented 23%. Seventy-one percent of respondents are from North American companies; 22% represent European companies, and 7% are from Asian Pacific companies. The participating companies have an average of 4.5 cell therapies and 3.6 gene therapies in their pipelines. Seventy-four percent stated using mid-size, full-service CDMOs followed by specialty (contract) labs at 58 and large, global CDMOs at 57%.
ISR asked, “Which therapeutic areas are the cell and/or gene therapies in your company’s pipeline designed to target?” (n=100), the data show that oncology (67%) is the most targeted therapeutic area for the cell and/or gene therapies in respondents’ company’s pipelines. Immunology (39%), hematology (28%), and neurology (21%) follow distantly. Indeed, ISR’s Cell & Gene Therapies Manufacturing Market Outlook (2nd Ed.)’s findings corroborate much of what we hear regularly from Cell & Gene readers as well as the subject matter experts who contribute guest articles and who appear as guests on Cell & Gene: The Podcast and as expert panelists on Cell & Gene Live events. For example, in this recent article on Cell & Gene pertaining to stem cell manufacturing, the author explains that stem cells are being studied for their potential to be used in different types of cancer like bone cancer, blood cancer, prostate cancer, or any other cancer therapies, such as developing personalized cancer vaccines and regenerating damaged tissue after chemotherapy. Stem cells have been used in bone marrow transplantation for treating blood disorders, such as leukemia and other blood-related cancers. And, they have shown potential in treating neurological conditions such as Parkinson's disease, Alzheimer's disease, and spinal cord injuries.
ISR’s report shows that in the next 18 months, the most frequently outsourced cell and/or gene manufacturing capabilities among respondents will be viral vectors (52%) and autologous cell therapy (49%). A slightly larger proportion of respondents anticipate outsourcing the manufacturing of viral vectors (58%), allogeneic cell therapy (45%), and mRNA (38%) in 5 years.
The most outsourced cell and/or gene manufacturing activities are process development / scalable manufacturing process (55%) and fill-finish (55%) among respondents. Other frequently outsourced activities include clinical supply logistics (46%), analytical development (45%), formulation (44%), and CMC (42%). ISR reports that those activities are anticipated to be outsourced at similar levels 5 years from now, except for fill-finish, which will decrease slightly. Fewer respondents outsource allogeneic cell therapies in comparison to autologous (38% vs. 49%). A larger proportion of respondents anticipate outsourcing allogeneic therapies five years from now (45%) than at present (38%).
Under viral vector outsourcing needs, respondents state lentivirus (58%) followed by AAV (48%), adenovirus (42%), and oncolytic virus (17%). For allogeneic cell therapy outsourcing needs, respondents listed cell-based immunotherapies (79%), iPSC manufacturing (37%), exosomes (5%), and other (8%). And for autologous cell therapy outsourcing needs, viral modification (65%) and non-viral modification at 43% and other at 4%.
When it comes to the primary outsourcing drivers for CGTs, ISR reports that 52% of drug developers engage CDMOs for cell and/or gene manufacturing activities to augment the supply they can manufacture in-house. Twenty-four percent of respondents shared that they have no manufacturing capacity / outsource 100% of manufacturing, while 16% indicated they have no cell/and or gene therapy. When asked, “About how many CDMOs do you think your company will need to meet your cell and/or gene therapy manufacturing needs five years from now?” (n=100), 40% of respondents stated they currently need two CDMOs, and that percentage drops to 19% in 5 years. Twenty-five percent of respondents currently use 3 CDMOs and 24% expect to use 3 CDMOs in 5 years. Fourteen percent of respondents stated they currently use 1 CDMO with 15% predicting they will use 1 CDMO in 5 years.
According to 26% of respondents, the primary reason service providers lose bids for a cell and/or gene therapy manufacturing project is “lack of experience with the type of therapy we are developing.” This is followed by regulatory violations (18%) and high cost (16%). Indeed, for years, Cell & Gene’s readers have stated lack of experience, high costs, etc. as reasons why potential partners lose bids.
This summary is only the tip of the iceberg. ISR’s Cell & Gene Therapies Manufacturing Outlook (2nd edition) is a must-read for any CGT company interested in understanding how CGT companies partner with service providers and why in order to scale.