Key Takeaways from the American Society of Gene & Cell Therapy 2026

RareKids-CAN was pleased to sponsor two of our Clinical Trial Navigators, Cara Grobbecker and Faiza Khawaja to attend the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting virtually.  

Faiza Khawaja (left) and Cara Grobbecker (right) are both RareKids-CAN Clinical Trial Navigators. Clinical Trial Navigators are essential to RareKids-CAN’s operations, servicing as “boots on the ground” across participating institutions. They connect investigators with rare disease resources, facilitate information sharing across the network, support local study teams with pediatric rare disease clinical trial (PRDCT) projects, identify clinical trial opportunities, and contribute to metrics collection. 

The conference brought together researchers, clinicians, industry leaders, regulators, and patient advocates from around the world to explore the latest advances in gene and cell therapy.  

While the science continues to move forward at an impressive pace, both navigators noted that successful implementation will require equal attention to patient needs, long-term follow-up, and health system readiness.  

Gene Therapy is Advancing Rapidly, But Delivery Remains Complex 

ASGCT highlighted the growing potential of gene and cell therapies to transform outcomes for people living with rare diseases. As Grobbecker noted, “Gene and cell therapies have the potential to treat many patients across a wide range of rare diseases,” but significant challenges remain across development, manufacturing, approval, and delivery. 

Sessions emphasized that gene therapy development differs substantially from traditional drug development. Because many therapies are administered only once, precision design, manufacturing, delivery, and patient selection is critical.  

Researchers also highlighted the importance of treating patients early, before irreversible disease progression occurs.  

Families and Caregivers Must Remain at the Centre 

A recurring theme throughout the conference was the need to focus on outcomes that matter most to patients and families. Khawaja observed that pediatric gene and cell therapy trials are increasingly moving “beyond biological proof-of-concept toward outcomes that are meaningful for children and families.” 

Measures such as communication, cognition, mobility, adaptive function, and quality of life are becoming increasingly important when evaluation whether a therapy is making a meaningful difference in a child’s daily life.  

Conference discussions also highlighted the need for clearer education and support for families navigating eligibility testing, treatment decisions, and long-term follow-up requirements. As advanced therapies move closer to routine clinical use, ensuring caregivers have access to understandable, practical information will remain essential.  

Regulatory Approval is Only One Step Toward Access 

Both navigators identified regulatory and access consideration as a major area of discussion. Rare disease gene therapies often require innovative trial designs because patient populations are small and traditional randomized trials may not be feasible. Researchers and regulators are increasingly exploring approaches such as natural history controls, adaptive trials, and real-world evidence to support evaluation and approval. 

At the same time, approval alone does not guarantee access. Grobbecker noted that reimbursement processes, infrastructure readiness, and inequities across health systems continue to delay patient access to approved therapies. Regulatory pathways, reimbursement models, and healthcare delivery systems will need to evolve alongside scientific innovation to ensure therapies reach patients safely and equitably.  

Looking Ahead 

Together, these reflections reinforce that the future of gene and cell therapy depends on more than scientific breakthroughs. Long-term success will require patient-centred trial design, meaningful family engagement, flexible regulatory approaches, and healthcare systems capable of delivering advanced therapies effectively and equitably. As Khawaja concluded, “clinical readiness must be matched by community readiness.”