Matthew Durdy, CEO of Cell and Gene Therapy Catapult, spoke at the 2024 MESA conference, where he was joined by Bert Bruce of Pfizer, Nuno Fontes of Bayer, Magdalene Pedersen of BMS and Manny Simons of Eli Lilly.
Big pharmaceutical companies took action at this week's 2024 Cell and Gene Conference, as they aim to expand their influence in the industry. Large pharmaceutical companies have played a significant role in the cell and gene therapy space, according to industry stakeholders at the annual Cell and Gene Conference held this week in Mesa. A growing consensus suggests that resource-rich pharmaceutical giants have a unique advantage in the commercialization of cell and gene therapies, which poses challenges for smaller biotech companies. As a result, Big Pharma is shaping the future of this field.
This was a key focus of a panel discussion on Tuesday at the Meeting on the Mesa, led by Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, an independent UK organization dedicated to the development of the field. Durdy noted that more than a decade ago, the isolated community of cell and gene therapy developers believed that large pharmaceutical companies would never “understand” this emerging market. Now, stakeholders “are looking for Big Pharma to really take the lead and help us overcome the difficulties we currently face,” said Durdy. He pointed out that high development and manufacturing costs are among the reasons for Big Pharma’s involvement. One of the biggest challenges faced by cell and gene therapy developers is the lack of funding, especially for early-stage startups. Pharmaceutical companies are providing part of the funding.
In March, Capstan Therapeutics completed a $175M Series B financing round, backed by new investors like Johnson & Johnson, as well as existing investors Bayer, Bristol Myers Squibb, Eli Lilly, Novartis, and Pfizer—all of which are eager to advance Capstan’s lead in vivo CAR-T candidate drug into early clinical proof-of-concept studies for autoimmune diseases. Last month, ArsenalBio raised $325M in a Series C round with new investor Regeneron and existing investor BMS, aimed at its solid tumor candidates using proprietary T-cell engineering technology.
Durdy also discussed how pricing and reimbursement pressures will require broader collaboration between the industry, healthcare providers, and insurers to deliver and scale life-changing therapies. “They have the potential to bring cures to patients.” Tim Hunt, CEO of the Alliance for Regenerative Medicine (ARM), echoed this sentiment during his welcome remarks on Monday, noting that 2024 has seen increased investment in cell and gene therapies. He pointed out that 13 of the top 15 pharmaceutical companies by market capitalization are “active” in the field. Hunt said many leading pharmaceutical companies—including Astellas, Bayer, Bristol Myers Squibb, Eli Lilly, Gilead, Johnson & Johnson, Novartis, Novo Nordisk, and Pfizer—are ARM members. ARM, a Washington D.C.-based advocacy organization representing the industry, hosts the annual Mesa conference, with several executives from these companies serving on its board.
With product patents expiring, major pharmaceutical companies face the dilemma of losing exclusivity and are looking to cell and gene therapies to fill the looming gaps. The U.S. patent for Bayer and Johnson & Johnson's blockbuster blood thinner Xarelto is set to expire in 2025. Nuno Fontes, Bayer's Senior Vice President and Global Head of Biologics Development, noted at the Mesa conference that the company's current clinical pipeline is split evenly between biologics and small molecules, with cell and gene therapies accounting for about two-thirds of its biologics pipeline. Astellas is also pursuing an early clinical pipeline in cell and gene therapies. Its Chief Scientific Officer, Yoshitsugu Shitaka, announced during a fireside chat at Tuesday's Mesa meeting an exclusive licensing agreement with London-based AviadoBio for its investigational gene therapy in Phase I/II development for patients with frontotemporal dementia caused by progranulin mutations.
“We expect this program to contribute to our revenue in the coming years,” Shitaka said. Under the Tuesday deal, Astellas will make a $20M equity investment and pay AviadoBio $30M upfront. AviadoBio is also eligible for potential licensing fees, milestone payments, and royalties, totaling up to $2.18B.
However, according to GlobalData, the number of deals related to cell and gene therapy in the pharmaceutical sector declined by 38% in Q2 2024 compared to the same period last year, and the number of patent applications in this area has also decreased. Nevertheless, the data analytics and consulting firm agrees with the sentiment expressed at the Meeting on the Mesa: Big Pharma’s interest in cell and gene therapy remains strong.
GlobalData concluded, “Despite the recent decline in patents and deal numbers, these technologies have the potential to significantly improve patient outcomes and expand market opportunities. Pharmaceutical companies are not only focusing on innovation to strengthen their patent portfolios but are also making strategic investments in cell and gene therapies.”
