Every year, the American Society of Gene & Cell Therapy (ASGCT) brings together the researchers, clinicians, and scientists driving some of the most important advances in genetic medicine. It is where the field measures its progress, shares new discoveries, and, at times, finds answers to questions that have remained unresolved for decades.

At Myrtelle, ASGCT is a moment to reflect on questions that drive every experiment, every clinical trial, and every conversation with a family who said “yes” when the outcome was uncertain.

And one question persisted above all others—one that researchers, clinicians, patients, and families continued to ask:

Does the gene therapy last?

The Finding That Changes the Standard of Proof

Durability is one of the defining challenges of Central Nervous System (CNS) gene therapy. Delivering a corrective genetic sequence to the brain is a feat of precision biology, and the field has questioned dose longevity. 

At ASGCT 2026, Dr. Paola Leone, Scientific Advisor to Myrtelle, presented data that finally answers the question. For the first time in the history of CNS gene therapy, persistent AAV cassette detection 15 years after gene transfer has been documented. 

This is the field’s first direct, long-term evidence that a single administration of CNS gene therapy can achieve truly durable, lasting biological correction.

“For years, families affected by this devastating leukodystrophy had no hope. Today, we are beginning to witness meaningful clinical changes that reinforce the potential of targeted gene therapy approaches to restore myelination and neurological function. — Dr. Paola Leone, ASGCT 2026

The Story Behind the Data

In 1996, Jordana Holovach, Myrtelle’s VP and Head of Communications, learned that her son, Jacob, had Canavan disease. Determined to find hope where none existed, she connected with Dr. Paola Leone, a gene therapy researcher whose work had initially focused on Parkinson’s disease. After meeting families living with Canavan disease, Dr. Leone committed her research to the disorder, helping to lay the foundation for the work that continues today.

 

At the time, the medical community’s opinion of gene therapy was clear: it was viewed by many as speculative, unproven, and unlikely to succeed. Families were urged to be cautious. Jordana heard those warnings. She understood the risks, the uncertainty, and the skepticism that surrounded the field. But she also understood what was at stake. 

In the early 2000s, Jacob became one of the first patients in the United States to receive an AAV gene therapy for a neurological disease.

 

The 15-year cassette detection data presented at ASGCT 2026—the finding now capturing attention across the gene therapy field—is not just a scientific milestone. It is deeply personal. Jacob’s treatment is part of that story.

The very therapy that many once questioned is now providing evidence of remarkable durability, demonstrating that the potential envisioned decades ago has endured.

What This Means For Families, Clinicians, and Investors

The weight of this durability data ripples across every sector of our community:

  • For Clinicians: 15-year persistence data is a massive material signal. It confirms that the oligodendrocyte-targeting platform underlying MYR-101 is structurally durable. 
  • For Researchers: The same platform being developed for Canavan disease is currently being applied to Pelizaeus-Merzbacher Disease, H-ABC, and Multiple System Atrophy. This data strengthens the scientific foundation beneath all of it.
  • For Investors: This finding addresses one of the market’s most persistent questions about CNS gene therapy scale. This is the kind of biological evidence that fundamentally validates the value of a gene therapy investment.

What’s Ahead

The momentum from ASGCT 2026 carries directly into Myrtelle’s regulatory path as BLA preparation continues. Our position in the FDA’s START Pilot Program enables direct, ongoing communication with the Agency on MYR-101’s development, while our RMAT designation supports eligibility for accelerated approval based on surrogate or intermediate endpoints and allows the option for rolling submission and review of BLA sections. 

To Everyone Who Refused to Give Up

Our deepest thanks to Dr. Leone, and to every collaborator who has spent decades in service of this work. To the Cure Canavan Fund, the Canavan Foundation, and NTSAD, whose support for families and grassroots research funding has been indispensable at every stage.

Together, we are working to make the “rare” seen, heard, and healed.

Stay connected with the Canavan community through the Cure Canavan Fund, the Canavan Foundation, and NTSAD. Follow Myrtelle on LinkedIn for real-time updates.