Btobioinnovation.com 

Author: Jean-Claude Muller, Executive Editor at BtoBioInnovation  jcm9144@gmail.com

SPECIAL REPORT 25.13

Cell and Gene Therapies: A Perspective

In the recent advertising of the GenScript Biotech Global Forum, to be held in London on November 20^th 2025, the text reads: “Cell and gene therapies (CGT) are advancing rapidly and are reshaping the future of medicine. From CRISPR-based genome editing to new applications of in vivo and ex vivo cell therapies, scientific breakthroughs are increasingly moving from the laboratory into clinical practice. At the same time, global investment and innovation in the field are accelerating, positioning CGT as one of the fastest growing areas in life sciences”.

This unbridled enthusiastic statement is, on the one hand correct but also challenged by some most recent events such as:

On September 24^th, 2025, uniQure (Netherlands) showed that AMT-30, a high dose gene therapy, administered by surgical procedure, in 29 patients, was able to achieve a 75% disease slowing of Huntington’s disease progression at 36 months.

Conversely, Galapagos (Belgium), Takeda (Japan) and Novo Nordisk (Denmark) have announced, over the past weeks, their decision to end programmes in cell therapy and are looking for divesting a business in which they had invested hundreds of millions of dollars. In all three cases, the companies had also announced restructuring of their businesses for financial and strategic reasons to explain their exit of ex vivo cell therapy.

This paper intends to be specific why advancement of these technologies is not as fast as recombinant proteins or monoclonal antibodies in the last decades. We have identified a series of key challenges and specific hurdles:

Manufacturing and Quality. Ex vivo cell therapies require an unusual apheresis process which consists in withdrawing blood from a patient, separating the desired cells while the remaining is returned to the blood stream of the patient. Cells are modified by various technologies and reinjected into the patient at a highly specialised facility. In case of an autologous treatment, the patient receives the very same cells that were withdrawn from his blood. If the treatment is allogenic modified cells are available “off-the- shelf”. The decentralised autologous process has a very sluggish throughput which requires multi locations and difficult scale-up timelines at each site.  The challenge lies in ensuring consistent GMP compliance and digital traceability across several hundreds of clinical sites. Allogeneic platforms must assure that no adverse immunology responses are occurring and as such trigger unprecedented quality control requirements.

Pricing and Payer’s Access. The very high upfront costs (more than several $100 000 and up to $2 millions) of CGT products are just not aligned with the current payer’s model of annualised budget and plan attrition. “With a projected pipeline of more than 150 therapies and more than $40 billion budget impact estimated by 2030, payers are looking for clarity as to what criteria may be appropriate for managing these products acknowledging that more innovative financing models will be required” wrote Katerine Kirk, director of commercial development on Cardinal Health’s Advanced Therapy Solutions.  Securing coverage of most CGT therapies remains a significant access challenge primarily due to high costs and unique patient profiles.

Regulatory. There is a is specific requirement for CGT treatments. Patients with CGT therapies undergo a significant long-term follow-up (LFTU) procedure, mandated by all regulatory bodies to monitor for adverse effects and sustained activity for up to 15 years for gene therapies. The patient usually must stay close to one month at the site of treatment upon the therapy infusion and in many cases the treatment protocol requires the patient to remain within a predetermined travel distance of the administration site and later on frequent follow-up visits during months. According to Professor David Porter, MD, Director at the Center for Cell Therapy and Transplant at the University of Pennsylvania “Between 20 and 40% of all patients who are predicted to potentially benefit for cell therapies ever get referred”.

The Patient Journey is rarely mentioned. For an autologous ex vivo cell therapy a patient would have to potentially travel several times to a specialised apheresis site, sometimes located hundreds of miles from home, to receive his modified cell injection. The patient will have to travel a very first time to the place for the enrolment process consisting of: patient education, patient consent to treatment, laboratory and genetic testing, travel and lodging coordination and authorisation from the insurance (weeks to months of waiting). The patient will have to travel again at least one day before the apheresis and stay one other day for surveillance.  Two to three weeks later, he will come back to receive its treatment, become an inpatient for a few days and stay up to 30 days at the hospital or close to it.  The travel and lodging costs are not covered by insurances. All over several thousand out of pocket dollars. A patient survey pinpoints that: lack of transportation, associated social support, and financial constraints represent 85% of the reasons why patients are not compliant to follow-up appointments and report treatments outcomes. A fact very different from what is observed for more established cancer treatments.

Infrastructure Needs. In addition to the above-mentioned hurdles there is still one any company must consider: behind each cell and gene therapy there is a lot of hospital infrastructure needed, such as dedicated apheresis suites, patient bed availability for a few weeks, as well as training of staff to ensure appropriate and efficient enrolment processes. In a nutshell: high additional costs.

The Future of CGT.

Many companies may have all the pieces but are potentially not able to put them all together. No wonder if some, after a comprehensive assessment of all these hurdles, associated with a potential low return on investment, are now reconsidering their previous strategy. But this does not mean that investors and companies should not stay engaged in this field. Continued scientific innovation, particularly in in vivo gene edited cell-based therapies and improved allogenic platforms is expected to improve efficacy, safety, and scalability. The development of new business models, adapted reimbursement strategies and robust real-world evidence will be essential to ensure broader access and sustainable growth.

Hopefully the next GenScript Biotech Global Forum under the name “The Next Era of CGT is Here”, alongside the Jefferies London Healthcare Conference (November 17-20) will not only be focusing on scientific progress and clinical outcomes but also address all the landscape including the specific issues inherent to cell and gene therapies.

Paris, November 11, 2025

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