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Miguel Forte, MD, PhD
President, ISCT
MC4TX
Belgium

The value that cell and gene therapy can deliver to patients is incredibly significant, from potent efficacy to long term benefits and actual cures. Those working in the field know this, believe it, and often wonder why others still question it. We must understand that CGT sits within a large landscape involving many factors, challenges and alternative opportunities. It is worth remembering that within the CGT space, there is currently a high degree of uncertainty. 

We have recently witnessed a period of hardship for biotech in general and for CGT in particular. Public investment in research and development is seen at risk and there has been less private investment, and several companies have been struggling to survive let alone to invest significantly. Nevertheless, the reasons to believe in the opportunity of CGT exist and continue to expand.  

Rapid Success in Regulatory Approvals

On the regulatory front, approvals are happening faster with 7 therapies expected to have accelerated approval in the period of 2025 to 2027 versus only 3 from 1992 to 2024. The total number of regulatory approvals is also increasing with up to 17 possible approvals this year with a clear catchup in Europe, associated in part with faster and more successful product approvals as indicated in a recent paper where CGT products have a success rate of 18.5% compared with 7.3% overall (1). These approvals occur together with an intense clinical trial activity with, as examples, more than 600 clinical trials with CGT products in solid tumors, and an expanding number of ongoing clinical trials aiming at resetting, rather than just controlling, the immune system in autoimmune diseases consisting of more than 100 studies with CGT products from a total of about 380 overall studies (2). A large part of these studies in autoimmune diseases are being done as academic clinical trials and in new geographies beyond the US.

China for instance, is attesting the broadening of the approaches and highlighting a key opportunity in public-private partnership in the early stages of clinical development of new targets. From the industrial perspective, the biggest challenge has been the slow adoption of CGT products in treating patients on the market. This is a key target as it translates the final objective of addressing patients’ unmet medical needs with actual treatment and the return on investment, giving confidence on the potential and sustainability of the field. CGT scientific breakthroughs are starting to become a  commercial success with 2 blockbusters up to 2024, three potentials in 2025 and an expected 10 by 2030 – all within a market that should grow from about 8 billion USD in 2024 to nearly 55 billion USD in 2030 (3). This commercial increased success results from multiple factors including the expansion to more prevalent diseases, the technical improvements resulting in more effective and lower cost of goods manufacturing.

Overall, this should result in stronger confidence in the field and an improvement of financing with investors making faster decisions, being more comfortable with the risk in CGT and trusting the outcomes of the development of these  products as delivering strong therapeutic value to patients and benefits to multiple other stakeholders. At the same time, companies are looking for alternative financing modalities to enable the progression of their development activities like licensing and merger and acquisitions, reflecting to a certain extent the “buyer’s marker” conditions.

The Promising Future of CGT

New developments continue to make the field exciting and very promising. The search for the resetting of the immune system, with approaches from CAR-T to engineered MSCs but always combining cell and gene engineering, and the promise of in vivo gene therapy, are two very significant examples. The tantalizing story of KJ, the child born with severe carbamoyl-phosphatase synthetase 1 deficiency, was treated with a customized lipid nanoparticle-delivered base-editing therapy. It was achieved through the collaborative effort between the Children’s Hospital of Philadelphia, the Innovative Genomics Institute, Berkeley, Danaher, and others, making KJ the first patient to receive a patient specific gene editing therapy (4).  This strengthens the growing opportunity of a platform approach to individualized therapies requiring new models and approaches suitable for the technology and the fantastic value it can deliver (5).

Beyond regulation, the public sector attitude and the political agenda appears also quite intent in supporting the CGT field. This could translate the fact that the key decision-makers see significant promises of societal and patient-targeted value. Despite the challenging environment, we can see signals of strong intended support for CGT by the FDA and its leadership. On June 5, 2025, a significant session took place (6) with the participation of FDA leadership and a diverse representation of individual leaders and organizations in the field of CGT including representation from ISCT. The outcome of the discussion touched on the desire for increased regulatory flexibility, speed, adaptability and attractiveness within clinical trial execution and support, all the way to increased adoption and use of CGT products. A point to make, and discussed at the session, is the balance between good deregulation, based on adequate risk/benefit analysis and adequate supportive evidence, versus inadequate and patient endangering approaches of uncontrolled use of CGT products. Overall, the collaborative attitude and desire to foster CGT products towards patient benefit is welcomed and should encourage the field of progress ahead.

In Europe, we see also a strong desire from the political leadership in promoting biotech innovation and development overall with clear attention to the CGT field. Attention to the need to make faster decisions on clinical trials, flexible approach to regulation and promotion of translational efforts, including with public supported new forms of active investment are areas where the European Commission, the Member States and the Regulatory Agencies are focusing on improving the overall activity of patient focused research and development. The Pharmaceutical Legislation review and the “Biotech Act” together with other actions driven by the EU Commission aim at support that through targeted legislation (7).

Ways To Make Meaningful Change and Progress in CGT

It is important and our responsibility as active players in the CGT field to sustain realistic hope about cell and gene therapy for the strengthening of the field and the increase of value delivery to patients. There is not a more well-suited place to do so than at the ISCT Scientific Annual Meetings–the most recent one for 2025 held in New Orleans, is a clear example. The ISCT Scientific Annual Meetings are a unique opportunity to gather widely diverse and representative groups of individuals and organizations involved in all aspects of CGT translational activities. It gathers academic researchers, regulators, technicians, physicians, industrial companies and respective members as well as investors and non-profit and patient organizations. It is a unique moment of exchange. The program includes a translation path providing state-of-the-art information on CGT, a round table path where discussion and exchange of views and ideas are incentivized, plus abstract and industrial presentations.

ISCT 2025 had more than 2,600 attendees from 55 countries and nearly 600 abstracts with immunotherapy and MSCs as the leading topics. Of note is that over 80% of those attending indicated that the program was novel and up to date and the meeting provided great opportunities for networking. The next annual meeting will be held in Dublin, May 6-9, 2026!

We are passionate about the value of the work in CGT and what it brings to society, multiple direct stakeholders and above all, the patients. We, therefore, must have the obligation to carry the message of hope and realism, while making all our contributions to strengthen the value proposition in multiple ways–from science to communication. We must do so as individuals, and as a group. ISCT, as an organization and through its activities, is one of the places to do it!

References

1. Phares, S; Phillip, K; Trusheim, M.; Clinical development success rates for durable cell and gene therapies; Nature reviews drug discovery, February 2025
2. Bhandari, M; Smith, J.F.; Capra, E.; Yang, G. The race to reset autoimmune disease, Nature reviews drug discovery, May 2025
3. Source: ARM; EvaluatePharma Analyst Consensus Estimates (May 2025), Deloitte Analysis
4. Musunuru, K.; et all; Patient-specific in vivo editing to treat a rare genetic disease; NEJM, May 2025
5. Marks, P; Progress in the development of N-of-1 therapy; NEJM, May 2025
6. https://www.fda.gov/vaccines-blood-biologics/workshops-meetings-conferences-biologics/cell-and-gene-therapy-roundtable-06052025
7. https://www.europarl.europa.eu/thinktank/pt/document/EPRS_BRI(2025)772866

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