Cell and Gene Therapy Sector Overcomes Manufacturing and Investment Hurdles Amid 30% Funding Growth
核心洞察
Despite market challenges, the cell and gene therapy (搜索) (CGT) sector saw investments reach $15.2 billion in 2024, marking a 30% increase from the previous year, with approximately 3,000 developers and 2,000 clinical trials currently active.
Manufacturing and distribution remain significant obstacles for CGT advancement, with industry leaders highlighting issues including limited treatment center capacity, single-supplier dependencies for critical materials, and patient cell variability that complicates standardization.
Experts predict the FDA will approve 10-20 cell and gene therapies in 2025, with automation, robotic systems, and in vivo approaches identified as key technological innovations that could help overcome current sector challenges.
The cell and gene therapy (搜索) (CGT) sector is experiencing robust growth despite facing significant manufacturing, distribution, and market challenges, according to industry leaders who gathered at recent high-profile conferences.
Investment in the CGT space reached $15.2 billion in 2024, representing a 30% increase compared to 2023, Rita Johnson-Greene, Chief Operating Officer of the Alliance for Regenerative Medicine (搜索) (ARM), revealed at the 2025 Cell & Gene Therapy Summit hosted by Charles River Laboratories.
"I know that 2024 was a very tough market, and it's a very tough time for cell and gene therapy (搜索), but a 30% increase year on year from a dollars-invested perspective, that's extremely important to note," Johnson-Greene said.
The sector now encompasses approximately 3,000 developers and 2,000 clinical trials, reflecting year-over-year increases of 6% and 3%, respectively. Thirteen of the 15 largest pharmaceutical companies by market cap are currently investing in CGT development or commercialization, including AbbVie (搜索), Astellas Pharma, Bristol Myers Squibb (搜索), Novartis, Roche, and Vertex Pharmaceuticals.
Manufacturing and Distribution Challenges
Despite the investment surge, industry leaders at the 2025 GenScript Biotech (搜索) Global Forum highlighted persistent obstacles in manufacturing and distribution that continue to hamper broader adoption of these therapies.
"The products are delivering, and the products are demonstrating the innovation for patients," noted Alan Bash, president of Legend Biotech. However, he emphasized that "challenges that we are experiencing are not the capacity constraints for internal manufacturing, but the capacity constraints of the treatment centers."
Jonathan Esensten, senior advisor at Multiply Labs and director of the Advanced Biotherapy Center at Sheba Medical Center, pointed to fundamental manufacturing limitations: "Current manufacturing often relies on 'classified clean rooms,' where people with their hands are actually manufacturing these products."
While automation and standardization offer potential solutions, Hari Pujar, operating partner at Flagship Pioneering, highlighted an inherent challenge: "No matter how much you automate, how much you standardize, you end up in the ex vivo situation with one variable, which is the patient cell."
Supply chain vulnerabilities compound these issues, with many critical materials, reagents, and equipment having single suppliers, creating risk of delays. Starting material quality inconsistency presents another significant challenge.
"Your drug is only as good as what you start off with...The patient has been through many different therapies, so what they have left can vary drastically," explained Rey Mali, chief business officer at Accellix.
Investment Landscape Evolution
The investment environment for CGTs has undergone significant changes since the COVID-19 pandemic. Carl Schoellhammer, associate partner at DeciBio, noted that generalist investors who entered the CGT space during the pandemic are now withdrawing after discovering the complexities of medical innovation.
Unrealistic expectations have also affected market perception, according to Amit Agarwal, managing director at Deloitte: "The markets, in many ways, are expecting every single product that gets approved is in and of itself going to generate a billion dollars plus. It just isn't going to happen."
For companies seeking funding, timing remains critical. Carter Caldwell, program director at Penn Medicine Co-Investment Program, advised against waiting until financial resources are depleted: "You should give yourself at least a year—probably more like 18 months right now—to raise a real round of funding."
Carolyn Ng, partner and managing director at TPG Life Sciences Innovations, emphasized that despite market fluctuations, quality opportunities still attract investment: "If you have conviction in your programs, go out there and tell the story and try to sell it, because there really isn't a good time that we can predictably foresee."
Clinical Progress and Future Outlook
The CGT field has made remarkable clinical advances, particularly in CAR T cell therapy (搜索). Carl June, professor and director of the Center for Cellular Immunotherapies at the University of Pennsylvania's Perelman School of Medicine, highlighted that "since the first approval, approximately 50,000 patients have been treated with CAR T cells worldwide, primarily for blood cancers (搜索)."
June characterized 2024 as a breakthrough year for CGTs, noting significant progress in glioblastoma (搜索) treatments and early-stage therapies for autoimmune diseases (搜索).
Looking ahead, Johnson-Greene expressed optimism about the sector's trajectory. She referenced former FDA Commissioner Scott Gottlieb's 2019 prediction that by 2025, the FDA would approve 10 to 20 cell and gene therapies annually. With nine approvals in 2024 and eight decisions pending, with potentially 15 total applications expected this year, Gottlieb's forecast appears on track.
"I believe that the future of cell and gene therapy (搜索) is bright," Johnson-Greene concluded.
Technological Innovations and Global Collaboration
Industry experts identified several technological developments that could help overcome current challenges. Automation and robotic systems were highlighted as potentially transformative, along with in vivo approaches that could circumvent many existing obstacles.
As Pujar posed: "What if we could create the cell inside the body…in which case we kind of default all of the challenges that have been mentioned?"
Global innovation, particularly from the Asia-Pacific region, is increasingly influential in the CGT landscape. Caroline Stout, partner at EcoR1 Capital, noted: "The pace of innovation coming out of China and Japan is formidable."
Despite geopolitical tensions, cross-border collaboration remains vital. Josh Resnick, senior managing director at RA Capital Management, expressed hope that politics would not impede scientific progress: "We view the world as fundamentally an open source to solve really hard problems."
As the CGT sector continues to mature, industry leaders emphasized the importance of educating investors, regulators, and the general public about the value of these innovative therapies, particularly for patients with rare diseases (搜索) and limited treatment options.
"This is why we do what we do," Johnson-Greene said. "This is why we get up in the morning. This is why we work so hard, right? It's for our patients."