Expert Warns Of Looming 'Capacity Crunch' For New Cell And Gene Therapy Products

Capacity shortages are looming for manufacturers wanting to develop and commercialize cell and gene therapy products over the next five years, an industry survey shows. The key finding: these firms should quickly build and staff the manufacturing facilities they will need.

Despite major strides being made in developing new cell and gene therapies, the lack of manufacturing capacity and shortage of talent are two problems that may constrain the future growth of this industry. Management needs to be convinced that it is necessary to redesign facilities and that the investment is worth the cost.

So asserted Eric Langer, BioPlan Associates president and managing partner, on July 11 at the BPSA Summit in Washington. Langer was citing the findings of BioPlan’s latest annual “Report and Survey of Biopharmaceutical Manufacturing Capacity and Production,” published in April. BioPlan is a biotechnology and life sciences market analysis, research and publishing organization. The report surveyed 222 biopharmaceutical companies and 130 industry suppliers in 22 countries.

Langer provided an overview of the market potential for these products, and described how a looming “capacity crunch” could delay commercial launches.

Strong Growth Potential

Langer said that the field of cell and gene therapies is surging as evidenced by the growing number of these therapies in development. There are about 1,000 of them in clinical trials. Even if just 10% make it through clinical trials to get approved, the sales of these products could generate $200 million a year. In five to 10 years, they could possibly generate yearly sales of $2 billion.

There are 12 cell and gene therapy products on the market today, and this is expected to grow to 80 in five years.

One attractive feature of these therapies is that insurance companies are willing to reimburse for these products because they cure diseases rather than simply treating them. They can cost up to $1 million and reflect manufacturing inefficiencies and high labor costs.

Yet manufacturers are confident that they will eventually reap the rewards of investing in these products. Gilead Sciences Inc. bought Kite Pharma Inc., a company that developed the chimeric antigen receptor T cell therapy Yescarta. Gilead seems assured it will eventually recoup its $12 billion investment.

Yescarta was approved by FDA in October for adults with relapsed or refractory large B-cell lymphoma. (Also see "Interview: Measured Launch, Long Future For Gilead CAR-T In Europe" - Scrip, 9 Jul, 2018.)

Langer acknowledged, however, that “it will take them a while to recover the profit from a $12 billion investment.”

Langer said that despite this potential growth and value of cell and gene therapy products, there is a looming “capacity crunch” that is expected to delay clinical projects and commercial launches. Firms must start investing in manufacturing capacity immediately. “If you don’t start today, [capacity] will not be there in five years.”

Innovators Using Stone Hammers

Langer said that to improve capacity, manufacturers need to redesign their facilities. They also need to invest in better analytics for in-house production, and use more closed systems as well as single-use systems, and invest in better autologous technology.

Langer said that “for every proactive manufacturer there are literally hundreds of innovators simply living with what they’ve got. They are using stone hammers instead of using much higher purpose equipment.”

Another challenge is the lack of trained staff with expertise in manufacturing. “This is all going to be a new and emerging area and it is going to require a lot of expertise.”

Respondents said they are having the most difficulty recruiting process development staff for upstream and downstream operations as well as process engineers..

Another problem is that existing staff may have the right academic background but not necessarily the manufacturing experience.

Langer said that “when you go into these facilities it will be like the ’80s where a lot of these people have academic backgrounds. These academics have never done this before. It will take time and keep in mind that they will have very limited industry experience.”

Capacity Crunch Will Affect Progress

Langer said that this potential capacity crunch is different than the one that affected the production of new therapeutic proteins and monoclonal antibodies 20 years ago. That capacity crunch, he said, has largely been resolved, while this shortage may not go away anytime soon.

In 2002, there was a capacity shortage for therapeutic proteins and monoclonal antibodies, as well as a lack of a qualified staff to deal with producing these products. (Also see "The Talent Challenge in Biologics Manufacturing" - In Vivo, 1 Jul, 2002.) Yet these problems have since been resolved with manufacturers expanding their capacity.

Forty two percent of the survey respondents said they expect to experience manufacturing bottlenecks within five years. Langer said that this capacity crunch means that manufacturers will have to wait 18 months before being able to launch cell and gene therapy products in a clinical setting.

More Manufacturing Centers

Langer said that a different mindset is also needed for cell and gene therapy products, one that is very different from the mindset for conventional drugs, and one that prioritizes short turnaround times.

“Remember one patient is waiting with their life for these cells to come back, this is what we’re talking about, they are not going to send them first-class mail. They are going to put them on a plane on a seat on a 747.”

Also, cells will be harvested and reinjected in a doctor’s office, and not in a “typical GMP facility.”

There is a need for more manufacturing centers to accommodate these shorter turnaround times. He said that “each product may require dozens of facilities worldwide.”

From the editors of The Gold Sheet.