Rapid advances in the development of cell and gene therapies over the past several years have heralded “an evolutionary time in medicine.”

That’s the opinion of Peter Walters, Director of Advanced Therapies at CRB. That thought reflects data seen in CRB’s annual Horizon Life Sciences report that highlights the rapid expansion of the development of cell and gene therapies across the industry. The data-driven CRB report finds biopharma leaders at a crossroads, facing “waves of change” in the mission to deliver lifesaving vaccines and therapies.

The Horizon report is based on survey responses from more than 500 industry leaders who provide insight into all corners of the industry. The investigation of cell and gene therapies is proving to be a disruptive space that is reshaping biopharma business models. When cell therapies first went into human testing, Walters said the big question on the minds of the scientists was, “will it work?”

“It was really a test of the science,” Walters explained. And, the science not only proved itself to be valid in the clinic, but in 2017, the U.S. Food and Drug Administration approved the first cell therapies aimed at hematologic cancers. The approval was so novel and historic that Scott Gottlieb, who served as FDA Commissioner at the time, was the one who made the public announcement heralding a new era of medicine.

“The CAR-T therapies have created a beachhead for a launch point of new companies,” Walters said, pointing to cell therapy companies such as Cabaletta Bio, Passage Bio and Century Therapeutics, Philadelphia-based cell therapy companies that launched in 2017 and beyond.

As cell therapy companies continue to push the boundaries of science and disrupt the industry overall, Walters noted that CRB’s 2021 report pinpointed a potential disruption within the cell therapy market. According to the report, nearly half of the respondents reported plans to move away from autologous cell therapies to focus on modalities with fewer challenges. Walters explained that in order to make cell therapies into a “robust scalable platform,” some of the cell therapy companies will evolve from autologous therapies to off-the-shelf modalities. He suggested that companies will take a hard look at using pluripotent stem cells that can be modified and then scaled up.

“A lot of companies are looking at allogeneic stem cells as a future-facing platform. It’s years out, but they’re looking to make this a key point in the future,” he noted.

While there is an indication that some companies intend to move away from autologous, Walters said that technology will remain in place for some time. He said that autologous cells are proven out to be more effective and longer-lasting than the allogeneic equivalents.

Walters added that manufacturing for this space will remain a paradigm of its own. He said the entirety of the manufacturing process must be performed with risk management throughout the entire process.

“Personalized medicine and the scale of it has flipped manufacturing on its head from testing, review, supply chain management, and staffing. Over the past few years, the industry has been struggling to innovate how to solve those problems,” Walters said. He added that these issues are challenging the industry’s historically conservative approach to such issues.

The CRB report also noted that a large majority of companies intend to continue using viral vector gene technologies in the near term to produce genetically modified cell therapies to ensure quality and reduce operational costs.

Another disruptive technology that will continue to play a significant role across the industry is the development of RNA-based therapies. The ongoing COVID-19 pandemic propelled the therapeutic approach of messenger RNA into the forefront of the public consciousness. According to the CRB report, nearly half of the respondents place significant emphasis on RNA-based therapies becoming a major portion of future pipelines in order to capitalize on speed-to-market and cost-of-goods advantages.

Walters said CRB has been keeping a close eye on the RNA space for the past few years. He said RNA was “one of those quietly-kept secrets” that CRB saw as having a huge potential.

“Then COVID hit and the world had the curtain turned back on RNA and now it’s a household term,” he said. Walters pointed to the rapid nature in which companies like Moderna and Germany-based BioNTech (which partnered with Pfizer) could map and design a therapy within hours of the virus’ sequencing shows the agile nature of RNA therapeutics. “It can pivot to handle a lot of different diseases very, very quickly.”

CRB anticipates seeing more companies shift into the RNA space in order to take advantage of the capabilities of the approach. For those companies that already had RNA therapies within their pipeline, the success of the technology will cause them to “double and triple down on it,” Walters added.

While the mRNA-based vaccines went through clinical testing at a breakneck pace driven by the threat of the ongoing pandemic, future research in this space for other disease indications will likely slow, Walters suggested.  

Other pieces of the CRB report include close looks at sustainability metrics within the industry, a focus on the development of oligonucleotides, speed to market, and the shift to Pharma 4.0, where companies will be able to operate digitally integrated facilities with predictive, real-time analytics. Report data shows that companies are aiming to improve their 4.0 capabilities over the next few years, Walters said.

“This is a tremendous report that pinpoints some of the looming, disruptive changes the industry is facing as it continues to move forward with its mission to deliver lifesaving vaccines and therapies,” Walters said.