2025 Outlook For CGT: Focus On Non-Oncology Indications, In Vivo Gene Editing, And POC
By Erin Harris, Editor-In-Chief, Cell & Gene
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The cell and gene therapy sector is poised for significant advancements in 2025 driven by three key trends: an expanding focus on non-oncology indications, the development of more precise gene-editing technologies, and the pursuit of scalable manufacturing solutions. As the field matures beyond its initial concentration on cancer treatments, researchers are increasingly exploring applications for genetic disorders, autoimmune diseases, and cardiovascular conditions. This broadening scope is complemented by the evolution of gene-editing tools, which offer enhanced precision and versatility in modifying genetic material. Simultaneously, the industry is addressing the critical challenge of scalability, with efforts to improve manufacturing processes aimed at making these therapies more accessible and cost-effective. These converging trends are likely to accelerate the development of novel treatments, potentially revolutionizing patient care across a wider range of diseases and bringing the promise of personalized medicine closer to reality.
But how? Companies such as Kiji Therapeutics, Orgenesis, and Precision BioSciences will play significant roles in shaping the field in 2025. These innovative biotechs are developing novel approaches to address unmet medical needs through advanced genetic and cellular engineering techniques. I caught up with leaders from all three companies to better understand their visions for their respective focus areas in 2025. Here are excerpts from my conversations with Kiji Therapeutics’ CEO and Co-Founder, Miguel Forte, MD, Ph.D.; Orgenesis’ CEO, Vered Caplan; and Precision BioSciences’ Vice President, Gene Therapy, Cassie Gorsuch, Ph.D.
A Focus on Non-Oncology Indications
I’ve had the pleasure of talking to Dr. Miguel Forte over the years about a whole host of CGT-related topics from tips for securing VC funding to ISCT’s focus in 2025 (Forte is President and Board of Directors chair of the International Society of Cell and Gene Therapy [ISCT]). When it comes to meeting the expected growing demand for cell and gene therapies beyond oncology in 2025, Forte explains, “A particular aspect is to be able to produce to meet demand of clinical indications with a significant number of patients at a competitive CoG that enables reasonable pricing. This is essential to provide access to a potentially large number of patients in need. Developing our MSC [mesenchymal stem cells] product sourcing the cells from iPSC [induced pluripotent stem cell] is geared at achieving that scalability.”
He explains that it is also important to do the product development with the commercialization and Health Technology Assessment (HTA) in mind to have a value proposition adapted to the launch’s needs. Finally, he suggests preparing all the aspects related to both the launch and the use of products from the early stage all the way to the moment of commercial launch, as that will be critical for the adoption and potential large use of these products.
In Vivo Gene Editing Advancements in 2025
Precision BioSciences’ Dr. Gorsuch expects in vivo gene editing to make significant strides as more programs advance to the clinic and produce meaningful data. Most will be in rare diseases aimed at delivering a gene knock out to drive a clinical response, but going into large patient populations will be the big advance and potentially transformative for the field. “Our program for chronic hepatitis B, which affects approximately 300 million people globally, is a great example as we aim to potentially cure hepatitis B by eliminating the root source of viral replication. I also anticipate advances in non-viral delivery, including lipid nanoparticles and novel AAV capsids. New delivery options could really expand the scope of gene editing to more complex diseases beyond the liver.”
As in vivo gene editing is still a relatively nascent space, Gorsuch shared that she expects regulatory frameworks will continue to emphasize stringent requirements for long-term safety, delivery platform validation, and off-target risk assessment. “Precision is developing substantial datasets in several models, including nonhuman primates to meet these requirements and, importantly, prioritizing early, transparent engagement with global regulators to align our preclinical packages with emerging guidelines. This early engagement means that we are prepared to do something that hasn’t been commonplace in gene editing: filing multiple regulatory applications nearly simultaneously and not waiting for clinical data from an early market before filing applications in others.”
Innovations in Point-of-Care Technologies
Just like my previous conversations with Dr. Forte, I had the good fortune to talk to Orgenesis’ Caplan back in 2022 about the business model for manufacturing CGTs at the point of care (POC). During that original discussion, Caplan shared ways to enable hospitals and healthcare providers to participate in CGT development by adapting them to closed system manufacturing rather than relying on the typical biotech model.
Cut to present day, and my most important question for Orgenesis’ Vered Caplan centered around the innovations in POC technologies in 2025 that may further streamline the manufacturing and delivery of cell and gene therapies. Caplan’s list of innovations includes miniaturized, automated bioreactors; AI-driven optimization; IT solutions; closed-system processing to prevent contamination, and new cell selection technologies. She shares that portable gene-editing tools and advanced quality control systems will improve precision and safety, and that modular platforms will allow easy adaptation for new therapies. “Together, these innovations promise to streamline CGT manufacturing and delivery, and we hope to see integrated innovation implemented in clinical settings,” she says.
It's no secret that the U.S. market faces real challenges when it comes to POC centers. Caplain explains, “U.S. POC centers for CGTs face additional challenges when compared to other markets. The regulatory aspect is the most prominent of these. The U.S. regulatory framework does not have the option of hospital exemption, and there is no clear regulatory pathway for decentralized production. The FDA, based on feedback from the industry and the healthcare system, is progressing. Major research centers run their own clean rooms. The cost of designing, building, and maintaining cleanrooms has become more and more expensive and requires knowhow and expertise that is difficult to attain and maintain.”
While challenges remain, Caplan feels there is potential for adoption in the U.S. “The basis for widespread implementation of POC supply of cell and gene therapy is a decentralized hub and spoke model supported with efficient IT solutions enabling comparability and central oversite,” she says. “Thus, integrating highly standardized processes (automation is important to achieve this), including analytics, training, production infrastructure, such as prefabricated GMP units as well as all other aspects of GMP can provide a solution. Each hospital developing new processes is economically inefficient and is difficult to control and regulate. Automation intended for a locally developed process is too high a financial burden, thus creating a dependency on a highly skilled workforce, which is in constant shortage.”
A Bright Future for CGT
“It is an incredible time for the genetic medicine field. As gene editing technologies, such as ARCUS, continue to advance in the clinic we are unlocking new possibilities to cure diseases by going after the root cause. We are excited to be at the forefront of medicine, bringing a new treatment option designed to provide a cure for people living with chronic hepatitis B.” Cassie Gorsuch, Ph.D.
