Myrtelle Launches Manufacturing of First-in-Class Gene Therapy for Canavan Disease in Strategic Alliance with Charles River and Viralgen

Team prepares for commercialization bolstered by START Program and regulatory recognition

NEW YORK, July 8, 2025 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a pioneering clinical-stage gene therapy company dedicated to revolutionizing treatment for neurodegenerative diseases, today announced the official launch of commercial-stage manufacturing for its first-in-class oligotrophic recombinant adeno-associated virus (rAAV) gene therapy product, developed specifically for Canavan disease (CD). This landmark achievement propels Myrtelle's therapy closer to market and patients who urgently need it.

The manufacturing program is being executed in strategic partnership with two global leaders in gene therapy infrastructure: Charles River and Viralgen Vector Core (a subsidiary of AskBio Inc.). Together, the trio is setting a new standard in viral vector innovation, combining Myrtelle's proprietary science with Charles River's GMP plasmid manufacturing and analytical excellence at their CDMO center of excellence in Keele, UK, and Viralgen's cutting-edge GMP vector production at their world-class facility in San Sebastián, Spain.

"This is a transformative moment for Myrtelle and the entire Canavan disease community," said Adrian Stecyk, Chief Executive Officer of Myrtelle. "Initiating commercial manufacturing represents the culmination of our bold vision—to translate deep scientific innovation into real-world therapies. With Charles River and Viralgen as our partners, we are poised to deliver a life-changing treatment with precision, scalability, and speed."

At the heart of this initiative is MYR-101, Myrtelle's pioneering rAAV-Olig001-ASPA gene therapy. Engineered with a unique tropism for oligodendrocytes—the myelin-producing cells compromised in Canavan disease—MYR-101 is designed to directly address the genetic root of the disorder by restoring ASPA enzyme function and supporting normal brain development. This targeted approach has the potential not only to halt disease progression but to enable true neural repair through remyelination.

"When every day, every hour, every minute makes a difference in saving lives, the Charles River team is excited and privileged to continue to support Myrtelle at this pivotal stage," said Kerstin Dolph, Corporate Senior Vice President of Global Manufacturing at Charles River. "Myrtelle's groundbreaking program exemplifies the progress gene therapy has made from concept toward commercial reality, and we're proud to be part of that journey, bringing hope to the patients and families affected by Canavan disease."

"Our mission at Viralgen is to transform people's lives through our manufacturing science," said Jimmy Vanhove, Chief Executive Officer at Viralgen. "Myrtelle's work in Canavan disease is extraordinary, and we are honored to contribute our expertise and advance GMP production to help make this therapy a reality."

Myrtelle's gene therapy program continues to gain momentum on multiple fronts. The Company remains on track to finalize regulatory filings and prepare for commercial launch, pending approvals. MYR-101 was selected as one of just four programs to participate in the FDA's Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program, a groundbreaking initiative to accelerate development of treatments for serious rare diseases by enhancing regulatory communication and flexibility.

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

• RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the U.S. FDA
• Orphan Drug Designation and ATMP classification from the European Medicines Agency
• Innovative Licensing and Access Pathway (ILAP) status from the UK MHRA

With manufacturing now underway in preparation for commercialization, Myrtelle is accelerating toward its goal of delivering the first approved therapy for Canavan disease, opening a new chapter in the treatment of rare neurodegenerative conditions—and offering a profound message of hope to patients and families around the world.

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 Myrtelle being poised to deliver a life-changing treatment with precision, scalability, and speed; Myrtelle's targeted approach having the potential not only to halt disease progression but to enable true neural repair through remyelination; Myrtelle's gene therapy program continuing to gain momentum on multiple fronts; Myrtelle remaining on track to finalize regulatory filings and prepare for commercial launch, pending approvals and Myrtelle accelerating toward its goal of delivering the first approved therapy for Canavan disease, opening a new chapter in the treatment of rare neurodegenerative conditions—and offering a profound message of hope to patients and families around the world. 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 deliver a life-changing treatment with precision, scalability, and speed; Myrtelle's targeted approach having the potential not only to halt disease progression but to enable true neural repair through remyelination and the ability to deliver the first approved therapy for Canavan disease; 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