Faces of Gene Therapy: Meet Allyson Berent

For Dr. Allyson Berent, science and motherhood collided the day her daughter was diagnosed with Angelman syndrome. Today, as Chief Science Officer at the Foundation for Angelman Syndrome Therapeutics (FAST), she’s turning that urgency into progress, advancing the first gene therapy programs for Angelman syndrome and reshaping how rare disease research moves from discovery to treatment. She also co-founded AS²Bio, a drug development accelerator that is building a new model for how science, philanthropy, and industry can work together to deliver treatments faster.

Allyson shared more with us about her journey.

Q: How did you come into patient advocacy and venture philanthropy?

I never planned a career in advocacy, but everything changed when my daughter, Quincy, was diagnosed with Angelman syndrome. I saw how much potential was locked inside the science – and how slowly the science was moving. I realized the Angelman community did not need another grant maker; we needed a builder. That’s what brought me to FAST.

In rare disease, waiting for someone else to take the lead can mean losing years. Venture philanthropy grew out of necessity. We raised catalytic funding, set milestones, and built what was missing so science could match families’ urgency. FAST launched AS²Bio, an accelerator that runs programs across gene replacement, stem-cell, and gene-editing programs under one coordinated ecosystem. MarviX Bio, a portfolio company of AS²Bio, recently achieved a major milestone: the first IND clearance for an AAV gene replacement therapy in Angelman syndrome, now in a Phase 1/2 clinical trial. Our job is to build, de-risk, and move credible options to families as quickly and safely as possible.

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

Angelman syndrome is a monogenic, non-degenerative neurodevelopmental disorder caused by loss of UBE3A function in neurons. By addressing the genetic problem at its source, you have the potential to change a person’s trajectory. That is why gene therapy matters. We see meaningful plasticity across ages in Angelman syndrome, so targeting UBE3A isn’t just theoretical. It has the potential to matter for children, teens, and adults.

For families, this means the possibility of durable improvement with limited dosing, which can lower day-to-day burden and create room for gains over time. Gene therapy won’t replace other paths – like antisense or small molecules – it will complement them. The goal is a full spectrum of options so every person living with Angelman syndrome can benefit, regardless of age or circumstance.

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

The biggest barriers aren’t just scientific, they’re structural. Translating discoveries into treatments takes funding, infrastructure, and time, and those are the resources most rare disease communities do not have. Even with strong science, regulatory studies and manufacturing are slow and expensive. It can take a decade or more and $20-50 million dollars before a therapy reaches patients.

Families do not have time to wait, and neither do we. Transparency and data sharing are essential, as is planning the regulatory path early and ensuring each program builds on what others have learned. When the field does this together, timelines shorten, costs drop, and safe, meaningful options reach patients faster.

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

Science alone won’t deliver treatments. Policy does. In rare disease, funds are limited, time is precious, and the benefit–risk calculus differs from common conditions. Angelman syndrome is not degenerative, but it is profoundly debilitating. Policy needs to reflect that reality and support approaches that can meaningfully change daily life.

Modernization means a few concrete things. Endpoints and biomarkers must reflect what families value and what regulators can rely on. Trials should be feasible for children, teens, and adults, with long-term follow up that does not overwhelm families. Coverage and payment models must be predictable so companies can invest, and patients can be treated. When those pieces align, industry participation rises and options reach people more responsibly.

As co-director of FAST’s Angelman Syndrome Biomarker and Outcome Measure Consortium (A-BOM), I see how true patient-focused development depends on this alignment. With momentum growing in Washington around gene therapy, the moment to act is now. Communities like ours can help policymakers understand what is meaningful to patients and caregivers and build policy that turns good science into care.

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

My hope is simple: that “rare” describes prevalence, not access. IGT can help by turning intent into usable rules: clear review paths, feasible trials, and coverage models that let payers say yes. We also need patient impact made visible through real-world outcomes, and investor confidence through predictable pathways and transparent data. If regulators, payers, sponsors, investors, and families align, meaningful options reach patients faster. Do this and “rare” stops being a reason to delay; it becomes a reason to deliver.