Alain Pralong1,3 Renaud Jacquemart2,3 Katrina Cordovado2,3
- Pharma Consulting ENABLE GmbH
- Omnium Global Consulting Inc.
- ENABLE Biotech AG
Biotech startups developing cell and gene therapies (CGT) often focus heavily on innovation—yet one of the biggest roadblocks to success is failing to plan for Chemistry, Manufacturing, and Controls (CMC) early enough. In fact, regulatory agencies like the FDA frequently issue clinical holds due to incomplete CMC data. How can you avoid these pitfalls and accelerate your path to clinical trials?
EARLY CMC INTEGRATION: AVOIDING REGULATORY PITFALLS
Start-ups developing advanced therapies may encounter significant obstacles when they neglect to incorporate CMC expertise early in their product development process. Without this critical knowledge, companies can face a range of CGT development challenges that can delay progress, prevent regulatory compliance, and jeopardize the success of their therapies.
- Product Quality Issues: failure to establish robust process controls early can lead to batch-to-batch variability, impacting safety and efficacy
- Regulatory Delays: inadequate or incomplete documentation and processes that fail to meet regulatory standards can slow down market approval
- Scalability Problems: difficulties in transitioning from small-scale laboratory production to commercial manufacturing
- Costly Setbacks: common pitfalls can lead to the need for extensive rework and additional validation studies
- Prolonged Time to Market: delays in development can extend the time required to bring products to market
For example, a biotech startup could face delays due to inconsistent product potency across batches. The lack of a well-defined potency assay and incomplete process validation data would result in a clinical hold or rejection of application, potentially delaying their trial by over 12 months, depending on the regulatory authority. To resolve this issue, the company would have to invest in additional assay development, comparability studies, and stability testing, increasing costs and extending their timeline.
In contrast, a company working with an experienced CDMO with integrated CMC support will be equipped to successfully navigate pre-IND discussions and proactively implement phase-appropriate CMC controls. This approach would accelerate their trial initiation and position them for a smoother path to commercialization.
Early CMC integration improves processes and ensures compliance with regulatory standards, allowing companies to bring their product to market efficiently and cost-effectively. A qualified CMC expert guides process validation, identifying and mitigating risks early to prevent costly delays and regulatory setbacks. Partnering with a CDMO experienced in CMC can streamline this complex process, accelerating the development timeline.
HOW ENABLE BIOTECH'S CDMO APPROACH GIVES STARTUPS A COMPETITIVE EDGE
ENABLE Biotech brings decades of combined experience to our clients. With an innovative setup for development and clinical manufacturing, we offer a unique approach tailored to the specific needs of each client at every development stage. One key pillar of this support is integrated CMC support, so sponsors don’t have to seek out external CMC consultants. Our network of highly qualified experts (CMC specialists, regulatory consultants, and bioprocess engineers) provide support with the preparation of CMC dossier sections while helping startups prepare for scale-up operations.
Unlike traditional CDMOs that treat CMC as a standalone service, ENABLE Biotech integrates CMC strategy into every stage of development, ensuring a seamless transition from R&D to clinical manufacturing:
- We don’t wait until IND filing; our experts proactively address scalability, analytical validation, and regulatory compliance from day one.
- Our team applies a phase-adapted approach that balances compliance with cost-effectiveness, avoiding unnecessary expenses while meeting regulatory expectations.
- Unlike rigid models, we offer both full-service CMC integration and a hybrid approach that allows clients to bring in their own experts if they prefer.
- With deep experience in cell & gene therapies, exosomes, and advanced biologics, we understand the unique CMC challenges of next-generation therapies.
- Our tech transfer process involves sponsor teams visiting our facility in-person to ensure a seamless transition of manufacturing processes from the laboratory to GMP conditions.
ENABLE Biotech believes in collaborating closely with our clients, offering early CMC strategy integration, flexibility, cost-conscious strategy, regulatory readiness, and deep CGT expertise. Our hands-on, customized approach to CMC is why startups trust us to help them de-risk development, accelerate time-to-market, and ensure regulatory success.
At ENABLE Biotech, we help CGT companies integrate CMC early to avoid costly delays and regulatory setbacks. If you’re preparing for IND or scaling up manufacturing, contact us today to see how our experts can support your journey.
REFERENCES
- “What Time Is Best to Bring My CDMO on Board?” What Time Is Best To Bring My CDMO On Board, April 17, 2024. https://www.outsourcedpharma.com/doc/what-time-is-best-to-bring-my-cdmo-on-board-0001.
- “The CGT CDMO Bottleneck Isn’t Capacity–It’s Capability.” European Pharmaceutical Review, June 12, 2023. https://www.europeanpharmaceuticalreview.com/article/183153/the-cell-and-gene-therapy-cdmo-bottleneck-isnt-capacity-its-capability/.
- “The Role of the Modern CMC Leader.” Division of Pharmacy Professional Development, April 1, 2021. https://ce.pharmacy.wisc.edu/blog/the-role-of-the-cmc-leader-in-the-outsourcing-model/.
