Myrtelle to Present Clinical Data and Regulatory Progress for MYR-101 Gene Therapy Program at ASGCT 2026

Myrtelle, Inc., a clinical-stage gene therapy company selected by the FDA for the START Pilot Program and focused on developing treatments for neurodegenerative diseases, has announced its participation in the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. Clinical trial data from its first-in-class, oligodendrocyte targeting investigational gene therapy program for Canavan disease will be presented at the event.

Dr. Paola Leone, an internationally recognized leader in Canavan disease research and scientific adviser to Myrtelle, will present in the session titled "Beyond neurons: Glial-targeted gene and cell therapies for neurologic and ophthalmic diseases," organized by the Neurologic and Ophthalmic Cell and Gene Therapy Committee. This session is scheduled for Tuesday, May 12th, from 3:30 p.m. to 5:00 p.m. ET. Dr. Leone’s presentation, "AAV Mediated Gene Therapy for Canavan Disease (Clinical Trial)," will focus on clinical data from Myrtelle's ongoing study.

"We're honored to present encouraging data from children treated to date at ASGCT, demonstrating signals of therapeutic benefit and meaningful clinical improvement, while contributing to the advancement of innovative gene therapy for Canavan disease," said Dr. Paola Leone.

Additionally, Adrian Stecyk, Chief Executive Officer of Myrtelle, will serve as a guest presenter at a lunchtime sponsored symposium on Wednesday, May 13th, hosted by Viralgen, Myrtelle's commercial manufacturing partner for MYR-101, the company's first-in-class gene therapy candidate for Canavan disease. The presentation will cover Myrtelle and Viralgen's collaboration, the status of Myrtelle's participation in the START Pilot Program, and the company's regulatory path toward BLA submission, including key data highlights from the gene therapy study.

"We are pleased to continue advancing MYR-101 in collaboration with Viralgen and to share our progress with the ASGCT community," said Adrian Stecyk.

The ASGCT Annual Meeting will take place from May 11 to May 14 at the Thomas M. Menino Convention Center in Boston, MA.

Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene, leading to a deficiency in the enzyme aspartoacylase. This deficiency impacts brain bioenergetics and development, affecting myelin production. Currently, there are no cures for CD, and only palliative treatments are available.

More information on Myrtelle's clinical trial in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by contacting Myrtelle directly.