Faces of Gene Therapy: Meet Amanda Moore

Amanda Moore is a devoted mother, advocate, and CEO of the Angelman Syndrome Foundation. After adopting her two sons, Jackson and Baden, in 2015, Amanda’s life took a transformative turn when Jackson was diagnosed with Angelman Syndrome, a rare neurogenetic disorder. Amanda has harnessed her experience and expertise in leadership development to elevate the voices of the Angelman community. Her work has brought together countless families through the ASF Family Conference, expanded access to care through new clinics, and raised national awareness with initiatives like the ASF Walk. Amanda understands that community, visibility, and early access to care can change the trajectory for families facing rare diseases like Angelman Syndrome. 

Amanda recently shared details about her advocacy journey and the importance of gene therapy for families like hers: 

Q: How did you come into the advocacy space? 

Amanda: My number one reason for coming into the advocacy space is my son Jackson who lives

with Angelman syndrome. 

Q: Why is gene therapy so important for your community specifically? 

Amanda: Gene therapy is incredibly important for the Angelman syndrome community because it targets the root cause of the condition, not just the symptoms. Where in most people, the maternal copy of a single gene: UBE3A is active in the brain, in individuals with Angelman Syndrome, the maternal copy is missing or not working, and the paternal copy, although present, is naturally silenced. Gene therapy offers the potential to either replace the missing gene or restore the silent paternal copy, restoring critical brain function. Early intervention is critical to better support healthy brain development and ultimately quality of life. This technology is new, and by revealing the brain's ability to adapt, new doors in the realm of gene therapy have opened up. For families, gene therapy represents so much more than treatment; it represents hope for a future where our loved ones with Angelman syndrome can walk, communicate, and live more independently than we ever dreamed possible. That’s why we advocate, support research, and show up every day. Because for the Angelman community, gene therapy isn’t just science it’s deeply personal. 

Q: What are some of the major barriers to access you see and how can we address them? 

Amanda: Insurance and Medicaid limitations often end with families facing denials or restrictions from vital services like behavioral therapy or specialized medical equipment. Later in life, there is a steep drop off in available support and services for adults with Angelman Syndrome. Through advocacy at both the state and federal level we can work to improve Medicaid waiver access, expand coverage for complex care, and require insurance to cover essential therapies and cover augmentative and alternative communication (AAC) devices. When we address these barriers and bring our lived experience to the table, that is when the real change happens.  

Q: As an IGT Advisory Council member, what do you hope IGT will accomplish? 

Amanda: I hope IGT will become a powerful force in breaking down the barriers between science, access, and families to accelerate the path from discovery to delivery of life-changing gene therapies. I think this can most effectively be done by first, championing equitable access and building a global framework ensuring fair, timely, and affordable access for all rare disease families. As a parent and an advocate, I know firsthand just how critical it is that lived experience shapes the science and I hope IGT continues to elevate the voice of families. Many rare diseases including Angelman Syndrome share challenges in delivery, regulation, and infrastructure. IGT can foster cross-disease collaboration to pool knowledge and resources so that progress for one, becomes progress for all. Cures only matter if we can deliver them and I hope that IGT will continue to advocate for policies and funding models that allow for long-term delivery, and manufacturing, especially in underserved communities and low-and middle-income communities. IGT has the potential to bridge hope with reality, and I feel honored to be a part of that effort. 

Q: Why do you believe IGT’s mission of modernizing the policy space for gene therapies is so important? 

Amanda: I believe IGT’s mission to modernize the policy space for gene therapies is not only important, it’s essential. As we enter a new era where science moves faster than policy, to families like mine, gene therapy represents what was once an unimaginable opportunity: the chance to treat the root cause of our loved one’s condition. But that opportunity means little if outdated systems, regulations, and access models keep those therapies out of reach. Modernizing policy is essential – access should not depend on your zip code, access for one needs to be access for all. Regulatory systems are not designed for one-time, curative therapies and we need new policy solutions that reflect the unique nature of these treatments. Families cannot afford to wait – time matters and every delay in access means lost developmental milestones and diminished quality of life. Policy should mirror the urgency that families live with every day. IGT’s work brings together science, policy, and the lived experience of families, that intersection is where meaningful, lasting change happens. I’m proud to support a mission that doesn’t just promise hope, but actively works to make that hope accessible, ethical, and equitable for all. 

Amanda’s advocacy journey is driven by the vision of a better future for her son, and the entire Angelman Syndrome community. Her voice is a powerful reminder that when science and policy work together, we can create a future where families impacted by rare genetic diseases have greater access to transformative care.