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From the President's Desk: Global Presence and Local Impact

  

Miguel Forte, MD, PhD
ISCT President
mC4Tx
Belgium

Dear ISCT membership as we come out of our annual meeting in Vancouver I take over as President and start my regular contributions to Telegraph. I aim to address topics that reflect the status of the field or relate to significant events, scientific, industrial or social. The articles are intended to be a dialogue between the membership and me with all in C&GT watching and reading!

The 2024 ISCT Annual meeting, in Vancouver, was a resounding success attesting once again to the leadership role of our Society in Cell and Gene Therapy translation, always with the patient in mind!

Through the four days, we had over 25 Translational Pathway Program sessions, 50 roundtables, 600+ abstracts and almost 2800 participants from 61 countries and over 7000 formal networking events. Attendance was evenly distributed across academia and industry, completing about 80% of participants, with non-for-profit and governmental just over 10%. This represents an increase of over 25% with about half of the participants attending for the first time!

A major aspect of the meeting was the very successful regulatory forum held on the Tuesday before the meeting, bringing together regulators from all over the globe to discuss the regulatory challenges and cooperation opportunities. The aim is to share the experiences and through that, calibrate regulatory risk assessment to foster regulatory decisions towards patient access. 

Indeed, patient access was at the top of the agenda throughout the meeting. Access starts with regulatory approval but requires capacity, both in terms of infrastructure and knowledge plus experience, on top of willingness to pay to enable patients to receive the needed therapies. We are still watching limited penetration of C&G treatments globally, with the majority of patients not getting access to treatments. ISCT, and I personally, have a focus on fostering access to evidence-based, regulatory-approved products. One aspect of that relates to the workforce training programs that ISCT is focused on delivering. A concern remains on access in less developed regions where infrastructure and financing represent a significant obstacle to C&GT products addressing unmet medical needs. I would like to promote the discussion around this topic and ways to address it throughout my presidency of ISCT. 

The roundtables, a hallmark of our annual meeting, were very popular, enabling intense and crowded discussions about many relevant topics. They represent a great opportunity for the audience to engage directly and straight into discussion mode with leaders in the field.

Also, in addition to the main program, both before the start and after the close of the meeting, several signature-series, focused registration-only discussion sessions, took place on topics from AI to MSC/exosomes development.

During the main meeting, the key topics covered included discussions on developments in CAR-T, MSCs, manufacturing, regulatory, industrial translation and funding. The hot topics are for sure the use of CAR-T in autoimmunity, the in vivo therapies, and we saw a lot of discussions on MSCs and exosomes, indicating a possible resurgence of these cells, in a more targeted way, back into the limelight of cell therapy!

These discussions and developments tell us about the maturity of the field with cell and gene therapy expanding into new areas and new technological solutions. In this context is important to manage expectations. C&GT can, and is, delivering enormous value to patients with opportunities for long-term, even cure benefits. Nevertheless, after fascinating initial results, we must be prepared, as scientists, academics, industrials and, very importantly, investors, for the need for endurance in product development, for significant costs and efforts in product development, that will translate to significant returns for patients and other stakeholders.

After the financing boom before the pandemic, biotech in general has experienced significant challenges in achieving funding in the last couple of years. This is only slowly, and in a shy way, improving as we saw in the discussions, on stage and off stage, at the meeting about the ability to raise. It is our joint responsibility to foster the right perspective on the value C&GT products are delivering, the stage of development we are and progressively enable access to these products for patients. That is what will bring value to the unmet medical needs and the return on the investment.

Creative and closer to the patients manufacturing options, with lower cost solutions, are the way to go to further enable the strength of the value proposition to all stakeholders. A lot of discussion on these topics, showing the improvements being made as well, highlights the critical need to continue to focus on this aspect.

It was also recognized that a large proportion of patients in need of these products still do not have access to them globally. Access is a combination of the capabilities to produce, administer and pay for them. ISCT has a lot to offer in terms of improving the capabilities, through the training programs, and has a vested interest in helping to find solutions to the affordability of these products for the benefit of patients. That will also support confidence in the field and foster investment.

During my presidency, I aim to strengthen the already well-recognized and active, global presence of ISCT. This global presence for local impact aims to be what consolidates ISCT as the leading organization in C&GT globally. Leading in translation, fostering patient access, globally, with a clear proactive and reactive ethic focus to fight against unproven cell and gene therapies and promote the right attitude towards the fantastic translation developments we are witnessing.

We all felt a fantastic atmosphere during the Vancouver 2024 annual meeting. With a great number of young, and promising, researchers there was a sense of discovery, excitement and sharing of knowledge, experiences and enthusiasm. It is that sense and feeling of partnership, joint objective, scientific rigour and willingness to share, learn and discuss that makes the meeting a great place to foster our activities, collaborations and value generation for patient benefit. It is a great growth opportunity for our early-stage professionals and a great forum for those already established to jointly enjoy the status of the field!

The atmosphere was not only scientifically charged, but it was also full of fun and friendship moments. The annual meeting is a moment of reunion of colleagues and friends. The Gala Dinner showed that and gave a clear sense of the youth of our well-established Society and a moment of great fun. It was also a moment of great food!

We must thank and acknowledge, first and foremost the ISCT Head Office team, but also the scientific organizers, the speakers, the exhibitors and sponsors and clearly the participants for the great success of the meeting! Now, together we all must navigate the year ahead, in translation and business, preparing and anticipating ISCT 2025 New Orleans in May 2025.

ISCT, we all, must repeat and augment this success in New Orleans. It is up to all of us to ensure that we grow in impact both for those attending and for the example and leadership ISCT brings to the field. The annual meetings are a clear example of a moment of reunion for our society and a moment to further the translation of C&GT products for the benefit and access of patients.


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