The Wild West. Like changing the engine of acar while driving down the highway.

This was how several cell and gene therapy industry leaders characterized the fields rapidly developing Biomanufacturing and supply chain environment at this years Maryland Tech Council (MTC) BIO Conference held in the BioHealth Capital Region (BHCR).

Cell and gene therapy is so new that itsmanufacturing and supply chain processes and best practices are stillcalcifying, leaving many organizations to learn on the fly as they attempt tobuild the efficiency and standardization necessary for the industry to trulytake off.

Put simply, cell and gene therapy companiesare doing something thats basically never been done before. Only a handful ofcompanies have successfully taken a cell and gene therapy product to market.Gene and cell therapy manufacturing and supply chain is truly a new frontierthat is just starting to be explored and mapped.

Earlier this year at the Bio Innovation Conference during a panel entitled, New Frontiers of Biomanufacturing, had a vigorous discussion about the evolving state of cell and gene therapy manufacturing and innovation. Panelists included Vigenes Chief Manufacturing Officer Jeffrey Hung, Ph.D.; Aaron Vernon, VP of Engineering and Supply Chain at Autolus; John Rowley, Ph.D., Founder, and Chief Product Officer at Rooster Bio; Chris McDonald, VP of Manufacturing at Kite Pharma and Robert Lindblad, Chief Medical Officer at Emmes Corporation, and was moderated by John Walker, Manufacturers Extension Liaison at NIIMBL (National Institute for Innovation in Manufacturing Biopharmaceuticals).

When asked, What keeps you up at night?,Jeffrey Hung of Vigene perfectly captured the conundrum facing those operatingin the cell and gene therapy field: What keeps me awake at night is thecontinued demand for clinical trial materials and commercial product while wekeep having to innovate at a fast pace. Its like trying to change a carsengine while driving it down the highway.

Other panelists cited what seemed likeinherent contradictions faced by a nascient cell and gene therapy industry. Inessence, these companies are learning on the fly without an established set ofrules to follow or even question. Production needs conflict with innovation;personalization is anathema to standardization; and being cutting edge oftenmeans they lack the tools, materials and the well-worn paths to regulatoryapproval already established in other biotech sectors.

As a supporter of manufacturerswhat we see is that everyone wants to innovate but at some point, you have to just bite the bullet and lock down your process to get reliable manufacturing techniques to move it along the regulatory pathway. Every tweak you do requires a lot of other work. You can work on innovation in the second stage of your product, stated Robert Lindblad of Emmes.

In the new frontier space, there are no reagents and no GMP reagents. You cant source GMP reagents so you have to qualify reagents just for your product and your indication, which is not adequate to get a certificate of analysis from the FDA. As you are on the cutting edge, you dont have the equipment to create a closed system, you dont have the reagents you need to have GMP manufacturing, so you have to be creative and work with the agencies to get through the regulatory pathways to commercialization, he added.

The personalized nature of cell and genetherapy also creates challenges for manufacturing standardization and supplychain. The one batch, one patient equation of autologous cell therapy makes ita unique and highly challenging manufacturing process.

When you think about designing and building aplant you cant build inventory. Biologic manufacturing allows for 2 or 3 yearsof inventory. For us, you can never take the plant down, stated Chris McDonaldof Kite Pharma. He added that in many ways building a cell therapymanufacturing plant is a lot easier than running one due to the challengespresented by personalization, constant production, lack of inventory and theoverall newness of the industry.

Rooster Bio has built its business model around solving some of the fields efficiency and standardization issues. Rooster is making great strides in its efforts to standardize parts of the manufacturing and supply chain processes by becoming the Intel of cell banking. By creating off-the-shelf, high-quality media and cells-similar in concept to what the Intel microchip did for the computer industry-Rooster hopes to help standardize an important segment of cell and gene therapy manufacturing process and supply chain, thereby increasing manufacturing efficiency while lowering the cost of cell and gene therapy costs to patients.

One bad reagent going into a cell bank thats supposed to last for a few years can be really debilitating. This is what makes the Rooster Bio business model possible. Innovation cant happen without quality. On the innovation side were in the middle of the process of living cells transitioning from being just a tool for research into technology itself, stated John Rowley of Rooster Bio.

He also cited Moores Law as an apt parallel for whats currently developing in the cell and gene therapy field right now. Moores Law states that the capacity of microchips would likely double every year while computers would decrease in cost. Rowley drew a link between Moores Law, the rapid increase in monoclonal antibody manufacturing capacity and cost reductions of the 1990s and what is going on now in cell and gene therapy manufacturing and supply chain.

While improving the manufacturing and supply chain is critical, Aaron Vernon of Autolus reminded the audience of the real-world impact of cell and gene therapy development failure or success. He emphasized the need for stronger cell and gene therapy manufacturing and supply standardization because of the direct link between personalized therapies and impacts on individual patients.

We have to have zero tolerance for manufacturing failures because of their direct impact on patients. There are a lot of moving parts and things get more complex over time. This doesnt scale easily, he stated. We want to innovate all the time but we dont decisions made early in the research process that hamper supply chain for a very long time.

Because personalized medicine is tailored for specific patients-i.e. one batch, one patient-the stake, while always high in biotech manufacturing, are higher in cell and gene therapy manufacturing and supply chain.

This makes solving the industrysmanufacturing and supply chain questions even more pressing. Having morecompanies successfully commercialize their cell and gene therapy products andincreased information sharing, even among competing companies, are critical tothe industrys future.

Theres a huge amount of knowledge out therebut theres a black box that only gives us information about whats workedand what hasnt really late in the game. We only learn from the FDA after thefact, stated McDonald.

Instead of relying on the FDA, Walker wonderedabout the possibility of sharing successes and failures among cell and genetherapies competing for market share.

Walker offered the following thoughts to thepanel, Different companies know whats working and whats not but because ofIP no one is sharing. As cell therapy is trying to move forward everyone istrying to protect their own space so they are not sharing failures. If youretrying to move the field forward scientifically thats one thing, but right noweveryone is thinking commercially and everyone is in their silo, which istotally understandable

Vernon noted that the Standards Coordinating Body and other organizations are working to develop manufacturing and supply chain standards for the industry and are actively seeking input from companies in the space.

What Ive learned more than anything recentlyis that these organizations need more industry engagement. There are certainthings-how we qualify shipping lanes, logistics, freezing, microbial testing,method validation-that are absolutely inefficient when we are reinventing thewheel all the time at different companies, stated Vernon.

Because this industry is so new-we only have4 or 5 approved cell and gene therapy approved commercial products-its reallyjust too early to be able to standardize, added Hung.

Because it is in fact too early tostandardize, cell and gene therapy organizations find themselves confronting aCatch-22. Manufacturing demand will compete with the need to innovate. Thepersonalized nature of cell and gene therapy will be at odds with the push tostandardize manufacturing and supply chain best practices. The push to beat thecompetition to market will inherently limit the data sharing necessary touplift the entire industry.

While these manufacturing and supplychallenges appear daunting, they always are when it comes to revolutionizingmedicine. Its the energy created by these contradictions that will driveprogress and foment innovation; its the immense challenges of frontierindustries like cell and gene therapy that will ensure the very best of thebest rise to success to pave the way forward for the organizations thatfollow. And its success that will breedmore success, as the conflict between these seemingly opposing forces will onlyresolve itself over time as more companies take therapies to market and thestories of their struggles and successes become public knowledge.

It seems like the Wild West now but conquering new territory is always complex and messy. The car will eventually have time to slow down and get in the shop to fine-tune its engine, offering a smoother, more efficient and faster ride to its destination.

The BHCR regions burgeoning cell and gene therapy cluster, as represented by those on the New Frontiers in Biomanfucturing panel, will clearly play a leading role in fulfilling the promise of cell and gene therapies to deliver high-quality therapies and cures to patients in need while driving down costs over time.

Learn more about working at Kite from Chris McDonald, VP of Manufacturing and Site Lead.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Biomanufacturing and Supply Chain Standardization Key to Success in Cell and Gene Therapy Industry Boom - BioBuzz