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

NEW YORK, May 11, 2026 /PRNewswire/ -- 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, today announced its participation in the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting where clinical trial data from its first-in-class, oligodendrocyte targeting investigational gene therapy program for Canavan disease will be presented.

Dr. Paola Leone, an internationally recognized leader in Canavan disease research and scientific adviser to Myrtelle, has been invited to present in the session "Beyond neurons: Glial-targeted gene and cell therapies for neurologic and ophthalmic diseases," organized by the Neurologic and Ophthalmic Cell and Gene Therapy Committee, on Tuesday, May 12th, from 3:30 p.m. to 5:00 p.m. ET. Her 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.

In addition, 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 highlight Myrtelle and Viralgen's collaboration, the status of Myrtelle's participation in the START Pilot Program, and the company's regulatory path toward BLA submision, 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.

ASGCT Annual Meeting: May 11 – 14th
Conference Location: Thomas M. Menino Convention Center, Boston, MA

Session: Beyond neurons: Glial-targeted gene and cell therapies for neurologic and ophthalmic diseases
Presentation by Dr. Paola Leone: Tuesday, May 12th, 3:30 p.m. – 5:00 p.m. ET Room 107ABC 
Sponsored Symposium Hosted by Viralgen: "A Platform's Path to Rare Disease Patients: Accelerating AAV Manufacturing from Development to Commercialization"
Presentation by Adrian Stecyk, CEO, Myrtelle: Wednesday, May 13th, 12:15-1:15 p.m. ET Room 107ABC

About Myrtelle
Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The Company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company's website at: www.myrtellegtx.com.

About Canavan Disease
Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene (ASPA) which prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain bioenergetics and development, including myelin production. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. 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 emailing [email protected].

View original content to download multimedia:https://www.prnewswire.com/news-releases/myrtelle-to-present-clinical-data-and-regulatory-progress-for-myr-101-gene-therapy-program-at-asgct-2026-302767509.html

SOURCE Myrtelle, Inc

[ Back To TMCnet.com's Homepage ]