The future of cell & gene therapy: Key trends to watch

Cell and gene therapies are moving beyond treating symptoms and towards correcting root causes of diseases, where a single intervention has the potential to last a lifetime. Despite manufacturing, scalability, and regulatory bottlenecks, experts in the field have pointed out certain trends that they foresee for bringing these therapies to millions. Let’s take a look at future trends in cell and gene therapy.

A significant trend in the cell and gene therapy space: the swing towards in vivo therapies  

A significant trend in the cell and gene therapy space is the swing towards in vivo therapies. In vivo, simply put, means inside the body. Cell and gene therapies are often developed ex vivo – outside the body – like CAR-T therapies for blood cancers such as Kymriah for B-cell acute lymphoblastic leukemia, Yescarta for B-cell lymphomas, Tecartus for mantle cell lymphoma and acute lymphoblastic leukemia, and Carvykti for multiple myeloma. There are in vivo treatments too, like Luxturna for vision loss, Zolgensma for spinal muscular atrophy, and Hemgenix for the blood disorder hemophilia B, albeit fewer in the market compared to ex vivo therapies. 

One of the reasons for this is that ex vivo cell and gene therapies have been deemed safer. Ex vivo therapies risk triggering fewer immune reactions than in vivo treatments. However, as advancements in vector engineering and precise targeting make headway, there could be an uptick in in vivo therapies.  

These treatments stand out, as they could greatly cut manufacturing costs and time. Ex vivo therapies remain vital, but experts predict that making changes to the genetic material inside the patient is becoming more popular. Moreover, while chimeric antigen receptor (CAR)-T cell therapies are famously ex vivo, as the CAR T cell is engineered outside the body and then reinfused, the in vivo way is to deliver genetic material directly to the patient. This genetic material, typically encased in a vector or a lipid nanoparticle (LNP) carries instructions for T cells to produce CARs that can identify and kill cancer cells.  

“There is a shift from manufacturing T cells in clean rooms (ex vivo) to directly engineering T cells in the patient (in vivo),” said Hendrik Dietz, chief executive officer of German gene therapy developer CPTx’s CEO. “This transition has the potential to bring cell therapies to millions of patients across multiple indications, including cancer, autoimmune diseases, and beyond. Billions are already being invested, though the winning architecture remains unclear. Alongside lentiviral vectors for integration and mRNA for transient expression, emerging DNA-based non-viral technologies may offer a more tunable in vivo solution.” 

CPTx is among the biotechs developing in vivo cell and gene therapies, and it has brought forward a vector platform different from certain established viral and non-viral delivery methods. Its immune-silent single-stranded DNA (ssDNA)-LNP platform does not permanently integrate with human DNA and, at the same time, does not induce a fleeting response. 

An ongoing debate is whether viral or non-viral vectors will take the center stage soon. A report titled Future Trends in Cell and Gene Therapies by Luis Pareras, neurosurgeon and founder of healthcare investment firm Invivo Capital, explained that traditional gene therapy relies on viral vectors, but these come with “baggage,” such as limited cargo sizes and high manufacturing costs. So, non-viral approaches – like CPTx’s – are having a moment. This method allows for repeated dosing, unlike viruses that cannot be redosed because of immune responses. 

Trends in cell and gene therapy: in vivo deals on the rise 

One sign that research and development (R&D) in cell and gene therapeutics is geared towards in vivo CAR-Ts is the growing number of deals and buyouts in the space. American pharma giant Lilly purchased Massachusetts-based Orna Therapeutics for up to $2.4 billion to have a stake in the field. Orna has created an engineered circular RNA platform for its pipeline of in vivo CAR-T cell therapies. 

Lilly followed suit with Gilead Sciences, which bought Interius BioTherapeutics for $350 million and Pregene for $1.6 billion, AbbVie’s $2.1 billion purchase of autoimmune-focused California-based Capstan Therapeutics, Bristol Myers Squibb’s $1.5 billion acquisition of Massachusetts-based Orbital Therapeutics, and AstraZeneca’s $1 billion grip on Belgian CAR-T developer EsoBiotec, all of which were signed in the span of a year. 

“In the past 12 months, multiple billion-dollar deals have signalled strong momentum toward in vivo cell engineering. The technologies are still young, with little clinical data and ample room for further innovation. Investors and partners will follow what is best for patients. We expect our platform to lead the next wave,” said Dietz. 

What’s going on with stem cell therapies? 

Meanwhile, the stem cell therapy market size is projected to cross $1.6 billion in the next four years. This is a 25.23% compound annual growth rate (CAGR) having reaped around $456 million in 2024.  

The approval of the first cell therapy Lantidra by the U.S. Food and Drug Administration (FDA) for type 1 diabetes in 2023 marked a milestone; being able to treat a chronic disease that affects hundreds of millions of people with a cell therapy. Soon, a stem cell therapy like Vertex Pharmaceuticals’ zimislecel could be the first of its kind to address type 1 diabetes. For diabetes, stem cell therapies work by replacing lost or damaged insulin-producing cells with pancreatic islets – a cluster of cells that regulate blood glucose levels in the pancreas. 

Treatments like this will start to become more widespread, according to Adam Hjorth, CEO of Nordic Stem Cell. 

“Within 10-15 years, we will see off-the-shelf stem cell therapies for large, underserved patient populations like erectile dysfunction (ED), diabetes, osteoarthritis, heart disease, and other cardiovascular diseases that have long been poorly served by current symptomatic treatments, among many,” said Hjorth, whose company is developing a stem cell therapy that uses a patient’s own cells to repair nerve damage to treat ED. 

With more than 8,400 clinical trials researching stem cells as of 2023, Hjorth believes that the future scale of the approach is “widely underappreciated.” 

“Barely hundreds of large-scale active stem cell clinical trials are approaching the point where results will be published,” he said.  

He added that this trend will materialize for both allogeneic, which uses stem cells from a healthy donor, and autologous, which uses the patient’s own stem cells.   

Hjorth said: “You can already see the early versions of this emerging, with trials covering all these areas, different approaches in development, including donor-derived allogeneic ADRC platforms, and many patents approved. Even when allogeneic therapies reach mainstream approval, a permanent market for autologous treatment will remain. A meaningful share of patients, particularly in the U.S., will not accept donor-derived cells for religious, cultural, or personal reasons. That segment does not disappear with scale.” 

This new line of biological stem cell drugs will be mass produced by big pharmaceutical companies and available at your local doctor’s office, ready for injection, he explained. 

“The therapies will be specifically developed and patented stem cells, grown in a specific way to be disease specific. In time, the treatments will become cheaper, more efficacious, and adopted at a mainstream level,” he said. 

“Two tracks are forming in cell therapy,” Hjorth added. One being the more formal approval pathway, “moving slowly, favoring scalable drug products for large diseases that have been poorly served by current symptomatic treatments.” The other being “real-world clinical practice, moving fast and already treating patients.” He anticipates that the gap between the two will “define the decade.” 

“The next decade will be defined by mainstream mass production of specifically grown stem cells for specific diseases, and that will be truly transformative. But not all patients can wait for that, which is why we see massive growth in high-end specialist clinics operating outside the E.U. and U.S.,” said Hjorth. 

Decentralized manufacturing models expected to improve scalability and access 

Joe O’Brien, vice president (VP) of Digital Transformation and Automation at Project Farma, sees this integration within healthcare facilities as a shift towards decentralized manufacturing models. 

“Producing therapies closer to the point of care has the potential to significantly improve delivery timelines and patient outcomes,” said O’Brien. 

Besides, now that the science is certain, the plan is to improve access to these life-saving therapies. 

“Efforts to reduce vein-to-vein time are becoming a central focus across the industry. Shortening this timeline not only improves manufacturing efficiency but also lowers overall treatment costs, which is critical to expanding patient access to these life-saving therapies,” said Skillin. 

Vein-to-vein time is the duration between the collection of cells from patients and the reinfusion of these cells once they’ve been engineered – particularly in the case of ex vivo CAR T cell therapies. According to J&J, the automation of manual steps in CAR-T manufacturing aims to address this. This can reduce “variability, minimize human error, and enable continuous operations,” according to the pharma giant. 

This comes with the escalating demand for CAR-T therapies, signaled by growing investor and big pharma interest. This underscores “continued confidence in both their clinical and commercial potential,” added Skillin. 

Overcoming hurdles to treat ultra-rare diseases; new initiatives on the way 

Current challenges in manufacturing, regulatory pathways, and access have made achieving clinical and commercial success tricky. For cell and gene therapies to treat rare and ultra-rare diseases, this is compounded by investor hesitancy. 

“Many promising therapies for ultra-rare conditions are being shelved, not because of safety or efficacy concerns, but because they do not fit traditional commercial models. This presents a business model rather than a scientific gap,” said David Barrett, CEO of the American Society of Gene & Cell Therapy.  

However, new projects intend to tackle this. For example, the American Society of Gene & Cell Therapy set up a task force focused on commercially pre-viable therapies. Along with Massachusetts-based Orphan Therapeutics Accelerator, it has formed CGTxchange, a non-profit that aims to create new treatment pathways and prevent trial pauses that disrupt patient care, often for children with ultra-rare diseases.  

“A more focused and discerning market is materializing, driving development of CGTs targeting more common conditions, while at the same time, we are seeing mission-driven approaches emerge, as traditional funding routes have become harder to rely on for ultrarare disorders. Rather than starting from scratch, there is growing momentum to keep existing therapies moving forward when programs are paused or abandoned,” said Barrett. “CGTxchange reflects this shift by providing a practical mechanism to connect shelved therapies with new developers and investors, helping reduce friction and expedite progress toward patients.” 

Orphan Therapeutics Accelerator’s CEO Craig Martin expects there to be continued movement toward distinct regulatory pathways for gene and cell therapies for ultra-rare diseases. 

“With some stumbles and learnings along the way, the application of plausible mechanism and platform designations will enable a higher number of treatments for ultra-rare conditions to be developed in parallel and more rapid succession,” said Martin. 

The cost of cell and gene therapies for ultra-rare diseases is also poised to go down, according to Martin, as there is renewed interest from the Asia Pacific region in the space. This could see a ripple effect in driving down costs in Europe and the U.S.  

“This will take some time, in particular given current global trade constraints, but the technologies emerging are compelling and necessary if we expect to make gene and cell therapies available to patients at the scale of need,” said Martin. 

Investors eye phase 2 success; dealmaking to be more selective 

Meanwhile, the current dealmaking climate reflects the emerging trends in the cell and gene therapy space. While deals are being forged, they have become more selective, Hjorth pointed out. Venture capital has dropped by 61% since the 2021 peak; nevertheless, the market has persevered, growing from $16.75 billion in 2024 to a projected $19.91 billion in 2025, with analysts projecting $91 billion by 2034, a report by BioSpace revealed. 

“Venture capital is concentrated at series B, around $60 million per deal in 2025. Big pharma operates differently: when a late-stage asset aligns with their portfolio, they pay a significant premium for the IP. There are many companies with trials coming out, and even a small team of 10 to 20 people can be sitting on extraordinary value after a positive phase 2 result,” said Hjorth.  

He thinks that an acquisition following phase 2 is far more attractive than raising capital for a phase 3 for founders and investors who are navigating FDA approval and chasing an initial public offering (IPO).  

“I expect many will take that path,” he said. 

He foresees that as cell and gene therapy developers with positive phase 2 data scout for buyers, competition in the future will be fierce. Comparing it to the GLP-1 hype, he believes that this will be the case for the first who holds positive results for large indications like ED, osteoarthritis, and heart failure, as these are cures, not merely symptom management. 

“Big pharma needs the IP, and sellers have every incentive to exit. They are scientists, not commercial organizations capable of rolling out treatments at a mass scale. I expect mass acquisitions across stem and gene therapy companies in the coming years,” he said. 

Despite regulatory pathways being complex and not always well suited to these innovative approaches, according to Barrett, scientific opportunity has also never been greater, with advances opening the door to treatments that were unimaginable just a few years ago. 

“This is where initiatives like CGTxchange play an important role by enabling new operating and financing models that allow nontraditional sponsors to step in and move programs forward,” said Barrett. “By pairing these innovative approaches with manufacturing and reimbursement strategies designed specifically for cell and gene therapies, there is an opportunity to turn assets that are currently considered nonviable into real treatments, reaching patients who might otherwise be left behind.”