Myrtelle to Present Breakthrough Insights on FDA's Groundbreaking START Pilot Program at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting in New Orleans on Thursday, May 15th

, /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a trailblazing clinical-stage gene therapy company on a mission to revolutionize treatment for neurodegenerative diseases, is proud to announce its participation in a highly anticipated panel at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting taking place on Thursday, May 15, 2025, at 3:45 PM CDT.  The session — "FDA's START Pilot Program in Action: Insights from Year One"—will spotlight Myrtelle's experience in this bold regulatory initiative designed to fast-track therapies for rare and life-threatening diseases.

This dynamic session will explore how the FDA's Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program is transforming regulatory engagement. The program enables more frequent and flexible communications between sponsors and the FDA—going beyond traditional meeting structures—to help accelerate the development of urgently needed treatments for devastating rare diseases.

Myrtelle's participation in START is already making a tangible impact on the advancement of its first-in-human gene therapy for Canavan disease (CD) — rAAV-Olig001-ASPA. The company was one of just three gene therapy developers selected by the FDA Center for Biologics Evaluation and Research (CBER) for inclusion. Through START, Myrtelle receives timely regulatory guidance and support, which is helping the company navigate clinical development with greater speed and clarity.

"The FDA's commitment to accelerating treatments for devastating rare diseases is not just inspiring—it's lifesaving," said Adrian Stecyk, CEO at Myrtelle. "By working side-by-side with companies like ours, they are helping to bring hope and momentum to communities that have waited far too long."

Myrtelle's gene therapy is uniquely designed to correct the underlying genetic cause of Canavan disease by delivering a functional ASPA gene using a proprietary oligodendrocyte-targeting AAV vector. This technology holds the potential to restore brain development and improve quality of life for children affected by this otherwise fatal disorder.

The company's progress has also been recognized globally, with multiple regulatory designations awarded to rAAV-Olig001-ASPA, including:

• RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA
• Orphan Drug Designation & ATMP classification from the European Medicines Agency
• Innovative Licensing and Access Pathway (ILAP) status from the UK Medicines & Healthcare products Regulatory Agency

Session Details

Date: Thursday, May 15th, 2025
Location: New Orleans Ernest N Morial Convention Center – Room 278-282
Time: 3:45 pm – 5:30 pm

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].

Forward-Looking Statements

This press release contains forward-looking statements. Words such as "may," "believe," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding the benefits to be derived from participation in the START pilot program including the program accelerating development of gene therapies for rare diseases that lead to significant disability or death within the first decade of life by facilitating more frequent advice and regular communication with FDA staff; reducing wait times associated with the formal FDA meeting process quickening the pace of development for products intended to address unmet medical needs; and the oligodendrocyte-targeting rAAV vector-based gene therapy restoring ASPA function and brain development in patients with CD. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelle's ability to derive the benefits from the START pilot program; Myrtelle's program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelle's expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

SOURCE Myrtelle, Inc