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Cell and gene therapies hold the promise of curing previously untreatable diseases. New scientific discoveries, technical innovation, and FDA approvals in CAR-T cell therapies and gene therapies are saving lives of patients suffering from diseases such as leukemia and sickle cell disease. However, those familiar with the field know that it’s filled with challenges. There’s no shortage of obstacles in the actual science -- ranging from developing the therapy itself to how you dose and deliver the drug to the patient. Always top of mind is the question of designing your process to be scalable and cost effective, ensuring that it is commercially viable and can actually get to the patients who need it.

Process development and scale-up requires a different perspective than research and discovery and has unique drivers. Experienced founders will tell you that one of the most challenging hurdles is incorporating a longer-term quality and manufacturing mindset at the earliest stages of making their therapy and developing their analytical methods. As start-ups build out their teams, it is important to incorporate diverse skillsets that will allow crucial design decisions to be addressed early on, saving time and money in the long run. Investors are interested in de-risked products, so it is important to have a strong understanding of your therapy’s critical quality attributes, process variables, and approach to scale-up. There is also a huge opportunity in using appropriate and cost-effective raw materials from day one! When patients are waiting for these life-saving therapies, you need to avoid redundancy in your development efforts as much as possible. We’ve been seeing more teams go into their entrepreneurial endeavor planning for regulatory and commercial success from the outset, either as the result of advice or experience.

Click here to read our previous blogs on Mitigating Regulatory and Supply Chain Risk.

ENTERING THE ENGINEERING MINDSET

Founders who have successfully navigated this shift reflect on adopting an ‘engineering mindset’. The research and development phase is no longer isolated. Instead, the focus is on integrating the various processes into a collaborative program thinking through logistics of getting your work produced at scale for clinical trials and eventually the market. There are a few ways you can tackle this step: outsource process development and scale-up to a specialty lab; bring on skilled personnel and invest in capital infrastructure to lead the work internally; or leverage a facility with shared resources to support your team in strategizing your next steps.

OPTION 1: OUTSOURCE

Outsourcing is always a solution for teams looking to expand their capabilities and capacity. It’s particularly appealing to small companies that may not be able to bring on a full-time employee or team to do the work. However, it still requires an investment of time and resources in conducting due diligence and building the relationship. Depending on the scope of work, contracts can require a significant financial commitment. Because cell and gene therapy is still evolving, contractors are continuing to build their experience with these technologies. It’s important for companies to be prepared to conduct tech transfer and put in the necessary amount of time to fully educate and align with their service provider. Be sure to factor these considerations into your budget and milestone planning.

OPTION 2: BUILD IT YOURSELF

When your therapy is complex and you want direct control, a common alternative is to build out your team and capex to support this work. This is a great option for companies that have the funding and leadership expertise to navigate the development and scale-up activities in-house. This approach provides the opportunity to nimbly address technical challenges and adapt to changing needs.  

OPTION 3: LEVERAGE SHARED RESOURCES 

Operating in a shared lab facility that offers equipment and expertise for early-stage process development provides the best of both worlds. LabCentral recognizes that outsourcing and hiring aren't always ideal strategies for early-stage life sciences companies at the forefront of cell and gene therapy innovation. To meet those founders’ needs, we developed specific capabilities to support this critical phase at LabCentral 238, one of our shared lab locations in the Boston/Cambridge area.

LabCentral's resident companies have the opportunity to mature in our network and de-risk their therapeutics using our specialized spaces and equipment for each stage of development. In addition to the traditional amenities and support, LabCentral 238 offers documentation write-up rooms and Process Development labs configured to support upstream bioprocessing equipment. LabCentral’s industry relationships help supplement staff expertise by providing opportunities for technical training and consultation tailored to the unique needs of their programs.

For example, LabCentral alumni, Satellite Bio, started at our 700 Main Street location and eventually moved into 238 Main Street at the beginning of establishing their drug substance and drug product manufacturing processes. For their technology, these processes involved isolation of cells from human liver tissue, cryopreservation, and pilot scale cell culture. Reflecting on their time here, Tom Lowery, Satellite Bio President and CEO shared, “By the end of our time at LabCentral 238 our process was developed, and we were regularly producing cryostable batches of materials at pilot scale. That same process is now on track to be used for clinical trial materials production.”

“LabCentral 238 was turnkey ready for our team. We had a fully outfitted tissue culture room that we were able to use for liver isolations, waste management support for our tissue waste, controlled rate freezers for cryopreservation development and gas plumbing to support about 10 cell culture incubators for our adherent cell culture,” Lowery, added. “Our team was able to focus exclusively on our process development and whenever we ran into needs with the facility, shared equipment or lab management the LabCentral team resolved the issue with the highest degree of professionalism. We could not have had that level of expertise and support for our infrastructure given the early stage of our company. “

Getting the right support for your team’s development process from the outset is critical to accelerating long-term growth and decreasing the time needed to get therapies to patients. If you’re interested in learning more about LabCentral’s capabilities and ability to support cell and gene therapy development, reach out directly at lrissin@labcentral.org, and check out our website to learn more. 

ASCGT 2025 ATTENDEES

If you’re at ASCGT 2025 in New Orleans, come find us at Booth #227 to talk all things cell & gene therapy! You can also schedule a meeting with our team in advance. Reach out to Lyndsey at lrissin@labcentral.org.