ASGCT Builds Bridges: Advancing Access Through Patient-Centered and Global Initiatives

At ASGCT's 28th Annual Meeting, the Society reinforced its commitment to patient-centered approaches through multiple sessions designed to elevate patient voices and address access challenges for cell and gene therapies (CGTs). 

Patient Perspectives in Development 

The symposium, To Biomarker Or Not To Biomarker? Use Considerations For CGT Drug Development, brought together regulators, researchers, and patient advocates to explore how biomarkers can be effectively integrated into clinical trials for rare diseases, while there are also cases where it may not make sense since there are many factors to consider. The session highlighted the critical importance of including patient perspectives when determining appropriate endpoints and outcome measures.  

Gain further insight on the use of biomarkers in CGT during this month's Community Quick Take, featuring Dr. Rebecca Ahrens-Nicklas. 

Advice to PAOs from The Business of Advocates Advancing CGTs 

This half-day workshop presented in partnership with the National Organization for Rare Disorders, provided practical guidance for patient advocacy organizations (PAOs) navigating complex CGT development landscapes. Speakers shared sustainable funding models and strategies that enable PAOs to effectively drive therapy development forward. 

Key advice included: 

1. Run your nonprofit like a business – Prioritize governance, fundraising, and strategic operations from the start.  

2. Choose experienced partners – Seek guidance and leadership from those with real-world, industry or pharma experience. 

3. Fundraise creatively and sustainably – Use diverse strategies, including venture philanthropy and capacity building grants (i.e. CZI Rare as One).  

4. Design trials around clear endpoints – Align with regulatory pathways and prioritize endpoints meaningful to your population. 

5. Secure your assets early – Negotiate licensing terms proactively to protect and maximize the value of your innovations.  

6. Invest funding to de-risk development – Focus on foundational tools like genomic data, AAV work, and toxicology to avoid clinical gaps. 

Addressing Access Barriers in the U.S. 

The symposium, U.S. Private Payment Challenges for CGTs, tackled critical post-approval access issues. Moderated by a member of the Government Relations Committee, the panel featured patient advocates along with a benefit strategy consultant to discuss how coverage policies impact patient access. 

The session explained how payment uncertainties create additional burdens for patients and providers, highlighting the importance of considering access challenges preemptively in therapy development. Panelists emphasized patient-centered approaches to coverage determinations and the need for streamlined navigation support for families seeking treatment. 

Expanding Global Access 

Complementing the focus on U.S. patient access, the Challenges and Opportunities for Developing Cell and Gene Therapies in LMICs symposium provided insights into expanding CGT access to underserved regions. The session, chaired by members of the Global Outreach Committee offered attendees insights into the regulatory considerations, clinical trial approaches, and localized manufacturing strategies that can accelerate CGT development in low- to middle-income countries. 

Speakers included representatives from NIH and Caring Cross discussing research readiness and regulatory environments; manufacturing experts highlighting localized production approaches; and academic leaders exploring collaborative models that enable rapid deployment of genetic therapies in resource-limited settings. The panel emphasized that global access requires tailored strategies that account for regional infrastructure, workforce capacity, and healthcare delivery systems. 

Building Community: Advocate Networking and Education 

Beyond formal sessions, ASGCT's Annual Meeting provided meaningful ways for patient advocates to connect, learn, and share experiences. Highlights included an impact wall in ASGCT Central where attendees reflected on what progress for patients means to them, zebra stickers to raise awareness of rare diseases, and an informal meet-up to connect with others advancing the patient voice in CGT. 

A standout moment was the Patient Advocate Connection Lunch, which brought together leaders from nearly 40 different disease foundations—creating a powerful, focused gathering amid the thousands attending the Annual Meeting.  

Participants chat at the Patient Advocate Conection Lunch during the 28th Annual Meeting.

The lunch began with a moving introduction from Sharon King, caregiver and co-founder of Taylor’s Tale, reflecting on her journey that began with raising research funds for her daughter—and came full circle 13 years later when she met the first child treated with the gene therapy her advocacy helped bring to life. Among the 60 attendees—primarily caregivers and patient advocates—were members of ASGCT leadership, including the Patient Outreach Committee. Each table was thoughtfully facilitated to encourage meaningful discussion, knowledge sharing, and a strong sense of community." 

These efforts build on the momentum of ASGCT’s commitment to patients, advocates, and caregivers and sets the stage for the 2026 Empowering Patients Summit, which will be held March 11–12. 

A Unified Vision for Patient Access 

ASGCT Board Member Allison Bradbury, PhD, (second from left) speaks with colleagues at the Patient Advocate Connection Lunch during the 28th Annual Meeting.

The insights and connections forged at this year's Annual Meeting will continue to inform ASGCT's patient engagement initiatives throughout the year, including the upcoming 2025 Policy Summit in September and the Empowering Patients Summit March 11 + 12, 2026. 

Caitlin is ASGCT's Public Policy Manager, Ali is ASGCT's Senior Patient Outreach Manager, and Lexi is ASGCT's Manager of Advocacy Programs.

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