The previous 12 months have seen considerable progress in cell and gene therapies, with the first solid tumor products receiving regulatory approval and applicants for treating diabetes and heart disease.
As CGT scale-up continued through the year, overcoming logistical hurdles, artificial intelligence (AI) helped optimize processes to ensure more patients could benefit from life-saving treatments.
These developments strongly indicate that the future is bright for the CGT market, which GlobalData forecasts will be worth $80 billion by 2030 — up from $6 billion in 2023.1 With so many breakthroughs in one year, the industry is in a strong position to increase the accessibility of treatments to more patients.
Advancements in the treatment of solid tumors and diabetes
Previously only approved for the treatment of liquid tumors, two cell therapies gained approval in 2024 for treating solid tumors. This advancement is significant, explains Nathan Frank, senior research scientist at Terumo Blood and Cell Technologies.*
“That we now have two therapies targeted at solid tumors is a big deal. The exciting thing about both these treatments is that they aren’t different iterations of the same therapy,” he says. “One is a TIL [tumor infiltrating lymphocytes], and one is a different type of TCR [T cell receptors], instead of a CAR-T. Subtle differences there, but both are novel and different.”
While GlobalData forecasts that oncology will dominate the CGT ma3rket, accounting for almost 45% of its value by 2030, research into other therapy areas will continue to expand through 2024 and after.1 Notably, a small study in China found the need for insulin therapy in diabetics was completely removed through the injection of autologous induced pluripotent stem cell (IPSC)-derived pancreatic islet cells.2
“The study is very small, so I hesitate to use the word ‘cure’ — but after a year, one patient still no longer requires insulin,” notes Frank. “That’s a massive deal.”*
Through injections into the abdominal tissue, the IPSC-derived islet cell product appears to engraft itself in the patient’s body and enable the secretion of insulin from the pancreas.
“This treatment will be allogeneic. So potentially, we won’t need to have the autologous therapy cycle in the future for diabetics,” adds Frank. “This could even be an off-the-shelf type 1 diabetes treatment. A patient wouldn’t have to undergo an intensive process — they could just receive treatment from a pharmacist. 2024 could be a really significant year that people look back on in terms of diabetes treatments.”
Overcoming logistical challenges with CGT
Current CGT costs are often in the hundreds of thousands of dollars per dose, which has been viewed as a major obstacle to the successful scale-up of the treatments. However, Frank sees reasons to be optimistic, as 2024 has also seen an improvement in infrastructure to support CGT deployment that will improve overall efficiencies in production and get treatments to patients more quickly.
“I know logistics aren’t as cool as technological advances, but they drive the world,” he explains. “You can have the best technology, but if you can’t get it where it needs to be when you want it, then it’s not worth as much.”
Frank describes the current lengthy vein-to-vein timelines for T-cell therapies as “unacceptable.”
“It’s a matter of two or three weeks sometimes, and in a lot of cases, especially when CGT is being used as a last-line therapy, the patient can actually pass away while the treatment is being manufactured,” he adds.
When CGT manufacture is centralized, the need for cryopreservation and other costly transportation drives up the final cost of the end-product. “Decentralized manufacturing is emerging as key — by producing the therapy at or near the hospital, you can ensure that no resources are wasted,” says Frank.
The role of AI in cell and gene therapy
Artificial intelligence is emerging as a key tool in the successful scale-up of CGT, helping to make development processes more precise. Notably, the FDA Modernization Act 3.0 — currently working through the U.S. House of Representatives as of November 2024 — aims to set standards for silico-organ modeling on a chip.
“They call them ‘IPSC village models,’ where you can explore how a particular drug affects all the different donor cells as opposed to using a cell line,” explains Frank. “From that, you get a far more subtle and nuanced picture of how that drug might react in human cells.”
These new ‘organs on a chip’ can, in turn, feed large datasets and AI models that generate real-time analysis of results, ultimately providing far more effective therapeutics for humans than traditional animal testing. “AI is helping move us closer to true precision medicine,” notes Frank.
What is on the horizon for cell and gene therapy in 2025?
As CGT know-how is applied to a greater number of diseases, and more treatments receive regulatory approval, Frank believes the future is bright for the industry. “More patients are being treated, and more money is being made. And that increases our ability to scale these therapies,” he says.
Frank predicts that autoimmune therapies will be a big trend in CGT for 2025, especially as researchers think bigger in tackling diseases with a larger patient pool, such as Crohn’s disease.
“One of the therapies approved in 2024 was for treating synovial sarcoma, which is amazing, as that’s a solid tumor. But it’s not a common cancer,” says Frank. “Looking at indicators with a larger patient pool will allow these things to move more swiftly towards approval.”
“In the future, I want people to be able to ask their doctors about cell therapy. I want people who aren’t in the industry to be talking about them and to be excited, to get that message out and encourage larger-scale adoption.”
Following such a milestone year for CGT, manufacturers in the field should seek expert partners to collaborate and build on the momentum to further the benefit of all patients.
References:
1. Pharmaceutical Technology. Cell and gene therapy approvals drive paradigm change in manufacturing. 14 October 2024. Accessed December 9, 2024. https://www.pharmaceutical-technology.com/features/cell-and-gene-therapy-approvals-drive-paradigm-change-in-manufacturing/?cf-view.
2. The Economic Times. In groundbreaking, world-first achievement, Type 1 diabetes cured in China using stem cell transplant. https://economictimes.indiatimes.com/news/international/world-news/in-groundbreaking-world-first-achievement-type-1-diabetes-cured-in-china-using-stem-cell-transplant-report-says/articleshow/113807844.cms.
3. Ash Clinical News. FDA approves two cell-based SCD gene therapies. February 2024. Access December 9, 2024. https://ashpublications.org/ashclinicalnews/news/7714/FDA-Approves-Two-Cell-Based-SCD-Gene-Therapies.
* Terumo Blood and Cell Technologies is not associated with these therapies.
