Large pharmaceutical companies were out in force at this week’s 2024 Cell & Gene Meeting on the Mesa, as they look to expand their presence in the industry.
Big Pharma has assumed an influential role as “power players” in the cell and gene therapy sector, according to industry stakeholders who attended the annual Cell & Gene Meeting on the Mesa this week.
The emerging consensus is that large, well-resourced pharma companies are uniquely positioned to commercialize cell and gene therapies, which can be challenging for smaller biotechs amid a myriad of hurdles. As a result, large pharma companies are shaping the future of the industry.
This was the focus of a Tuesday panel at Meeting on the Mesa led by Matthew Durdy, chief executive of the Cell and Gene Therapy Catapult, an independent U.K. organization committed to the advancement of the field. Durdy observed that more than a decade ago the belief among the insular community of cell and gene therapy developers was that Big Pharma was never going to “get their heads around” the nascent market.
Now, stakeholders are “looking towards Big Pharma to really take the lead, to take us over that hump that we’re in at the moment,” Durdy said.
He pointed to high development and manufacturing costs as one reason Big Pharma’s involvement would be of service. One of the biggest challenges that cell and gene therapy developers are facing is a financing environment where capital is scarce, especially for early-stage startups. Pharma companies are providing some of the funding.
In March, Capstan Therapeutics closed a $175M Series B financing round with backing from new investors including J&J, as well as existing investors Bayer, BMS, Eli Lilly, Novartis and Pfizer—all lining up to advance Capstan’s lead in vivo CAR T candidate to early clinical proof-of-concept studies in autoimmune disorders.
Last month, ArsenalBio raised $325 million in Series C funding—with new investor Regeneron and existing investor BMS—for its solid tumor candidates that use a proprietary T cell engineering technology.
Durdy also discussed how pricing and reimbursement pressures require even broader collaboration—between industry, healthcare providers and insurers—to deliver and scale life-changing therapies. “They have this ability to bring therapeutics to patients.”
Ebb and Flow of Deals
In welcome remarks at the conference on Monday, Alliance for Regenerative Medicine (ARM) CEO Tim Hunt said there has been uptick in investment in cell and gene therapies in 2024, noting that 13 of the 15 largest pharma companies by market capitalization have an “active presence” in the sector.
Hunt said many leading Big Pharma companies—including Astellas, Bayer, BMS, Eli Lilly, Gilead, J&J, Novartis, Novo Nordisk and Pfizer—are members of ARM, a Washington, DC–based advocacy organization that represents the industry and hosts the annual Meeting on the Mesa, with several executives from these companies serving on its board of directors.
Faced with a loss of exclusivity as product patents are set to expire, major pharmas are looking at cell and gene therapies to help fill the looming gap.
The U.S. patent for Bayer and J&J’s blockbuster blood thinner Xarelto will expire in 2025. Speaking at Meeting on the Mesa, Nuno Fontes, senior vice president and head of global biologics development for Bayer, said the company’s current clinical pipeline is evenly divided between biologics and small molecules—but within biologics, cell and gene therapies account for about two-thirds of its pipeline.
Astellas is also pursuing an early-stage clinical pipeline of cell and gene therapies. CSO Yoshitsugu Shitaka in a Tuesday fireside chat at Meeting on the Mesa announced an exclusive license agreement with London-based AviadoBio for its investigational gene therapy in Phase I/II development for patients with frontotemporal dementia with progranulin mutations.
“We expect that program to contribute to our revenue in coming years,” Shitaka said.
Under Tuesday’s deal, Astellas will make a $20 million equity investment and pay $30 million upfront to AviadoBio, which is also eligible for a potential $2.18 billion in license fees, milestone payments and royalties.
However, the number of cell and gene therapy–related deals in the pharma industry declined by 38% in the second quarter of 2024, compared with the same period last year, according to GlobalData, and there have been fewer cell and gene therapy–related patent applications from pharma companies. Nevertheless, the data analytics and consulting firm agreed with the sentiment here at Meeting on the Mesa: Big Pharma’s interest in cell and gene therapy is strong.
“Despite a recent decline in patents and deals, these technologies are poised to significantly enhance patient outcomes and expand market opportunities,” GlobalData concluded. “Pharmaceutical companies are not only focusing on innovation to enhance their patent portfolios but are also making strategic investments in cell and gene therapy.”
