The Power of Patient Voices: A Preview of the Empowering Patients Summit

The journey to developing life-changing therapies is not just about groundbreaking science or cutting-edge technology. It is about people. Patients, advocates, and caregivers bring essential perspectives to the table, shaping the future of treatments in ways others cannot. Their lived experiences, challenges, and hopes are the heart of the drug development process. Yet, true progress happens when these voices are heard alongside those of industry leaders, regulatory experts, and scientists. Collaboration ensures that therapies address real needs, improve lives, and reach those who need them most. 

This is why we are excited to highlight Empowering Patients 2025: A Cell and Gene Therapies Summit. The summit addresses educational needs expressed by the patient community and offers discussions to increase knowledge among patients, advocates, and foundation leaders so they feel empowered to have a seat at the table. 

The beginning of the event will level set the current science behind gene and cell therapy approaches. With many possible ways to target a disease or genetic variation, there are different gene and cell therapy strategies to consider. Understanding these nuances can aid in discussions when pursuing research toward treatments.

Watch the video below to hear from ASGCT President Paula Cannon, PhD, on the importance of having different types of CGT stakeholders, including patients, gathered together to learn from each other.

A main focus of the event will address clinical trials for rare diseases. CGT clinical trials require special consideration prior to design and implementation due to the often-small populations, the urgency for treatment, and the potential shorter pathways to approval. Eligibility criteria, dosage, and control mechanisms must be carefully determined. The summit aims to offer a deeper understanding of this decision-making process of clinical trial design and its impact on participants. Additionally, we'll talk about the goals and personal values that should be discussed early in the trial process. Managing expectations at every stage—whether just beginning, or on your way to seeking approval for a therapy—is key to navigating difficult conversations among advocates, participants, regulatory agencies, providers, and more.  

Clinical trials involving CGT approaches are still being researched, and many therapies that show promise may never get FDA approval. Sometimes we see commercially pre-viable therapies often lose momentum due to high costs, regulatory challenges, and small patient populations. However, potential solutions may exist, offering hope to patients and families in need.  

Join us to amplify these vital conversations and ensure all levels of patients, caregivers and advocates are benefiting from this opportunity to learn! Be sure to share among your networks and register today!  

Related Articles