Overcoming Key Quality Challenges in Cell and Gene Therapy: A Q&A with Rafael Torres
A few frontline insights on overcoming some of the most pressing quality challenges that CGT companies face today.
Cell and gene therapy (CGT) has rapidly emerged as one of the most promising fields in modern medicine, offering the potential to cure previously untreatable diseases. However, as exciting as the science is, the complexities and challenges of developing and manufacturing these therapies are equally daunting.
The path from R&D to commercialization is filled with regulatory hurdles, intricate manufacturing processes, and stringent quality requirements that must be meticulously navigated to ensure product success.
We sat down with Rafael Torres—a seasoned expert in CGT quality management—who shared his firsthand insights into overcoming some of the most pressing challenges CGT companies face today. Rafael provides practical, real-world solutions to these challenges, ranging from workforce retention to managing complex CMO relationships and contamination control.
Rafael has over 23 years in the pharmaceutical and medical devices industries. His tenure includes significant roles in multinational companies, where they led quality programs that drastically improved efficiency and reduced defects.
At Ultragenyx Gene Therapy and Oncologie Ltd., he ensured gene therapy products met the highest quality standards, complying with cGMP and GCP regulations. His efforts included managing process improvement projects, coaching, quality systems, and active engagement in developing and implementing regulatory standards for innovative gene therapy products.
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Q: How can companies retain workforce knowledge and manage the risks associated with staff turnover in cell and gene therapy projects?
This is one of the biggest risks I see in small biotech companies—especially in cell and gene therapy. These teams are small, often just 15 to 20 people, and each person holds a tremendous amount of knowledge about the product. I’ve seen companies where someone who has been with the team for five years leaves, and suddenly, all of that knowledge about the product’s raw materials, processes, and development stages is gone. The replacement might have similar qualifications, but they’re not going to come in with that specific knowledge, and that can lead to major disruptions in clinical or manufacturing phases. I had a client who lost their most important manufacturing person in the middle of phase II trials, and it set them back months.
Now, that turnover doesn’t just happen because of external opportunities. In this field, you’ve also got a lot of pressure—sometimes the work environment becomes toxic because companies are up against tight deadlines. I’ve seen cases where people are burned out from pushing to meet impossible timelines. You’ll have CEOs or shareholders demanding that the product hit the market by a specific date, and that creates a fast-paced, high-pressure environment that’s hard to sustain.
You’ve got to focus on knowledge transfer and employee retention strategies. First, document everything. And I don’t just mean the SOPs—sure, you need those—but also informal knowledge. Why certain decisions were made, what went wrong in trial runs, and how the team adjusted. You should be recording that in a way that’s easy for new people to access. One client of mine used a digital platform to store these decisions, including video walkthroughs and meeting notes, so the new hires could quickly get up to speed.
Mentorship is also key. You want your senior people passing that knowledge down to newer team members. If you’re not creating a culture where people are actively sharing knowledge, you’ll lose it the moment someone walks out the door. And look, in the early stages, you might not need a full-time replacement right away. Bringing in part-time consultants can fill that gap. I’ve seen it work really well—consultants come in with specific expertise in cell and gene therapy and can help maintain continuity while you build out your team.
Q: Cross-functional teams often struggle with understanding Good Clinical Practices (GCP). How can companies ensure all teams are aligned on compliance?
That’s a huge issue. I’ve seen it time and time again. You’ve got departments like IT, CMC, regulatory, and clinical all working together on the same project, but they’re not necessarily speaking the same language regarding compliance. One team might be hyper-focused on hitting their own goals but overlook the bigger picture of how their work fits into the regulatory framework.
For example, I’ve seen IT teams make changes to data management systems without understanding the FDA’s requirements for data integrity, or a CMC team tweak a manufacturing process without realizing it could put the entire project out of compliance. One company I worked with had this exact problem—they changed their clinical development without considering how it would affect their regulatory submissions. They had to go back and redo much of the work because they missed critical GCP requirements.
The first step is to provide cross-functional GCP training. Every department needs to understand how GCP applies to their specific role. It’s not enough for the regulatory team to handle it. CMC needs to know how its manufacturing processes impact clinical data, and IT needs to understand how to protect data integrity. Tailor the training to each department’s needs and show them real-world examples of where a lack of compliance derailed a project. I’ve seen companies use case studies where non-compliance led to regulatory delays—it helps bring the message home when people see how easily things can go wrong.
Also, it’s essential to create formal communication channels between departments. I always recommend setting up a quality review board with representatives from each team—IT, CMC, regulatory, and clinical. They should meet regularly to discuss any compliance issues or upcoming projects. This way, everyone is on the same page, and potential compliance risks are flagged early on.
Q: What are the key challenges in managing raw materials and CMOs in cell and gene therapy, and how can companies address them?
Managing CMOs and raw materials is one of the biggest headaches in cell and gene therapy. You’re often working with multiple suppliers, and it can go south very quickly if you don’t stay on top of things. I’ve seen companies waiting weeks for batch records only to find out that the CMO had an issue they didn’t report. By the time the company found out, it was already too late to meet their submission deadlines, and that’s a nightmare when you’re under pressure to get to the next phase of your clinical trial.