New York, NY May 7th, 2024 – Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical-stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, will host a symposium on Canavan disease (CD) clinical trial updates and future directions for oligodendrocyte targeting AAVs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting on Wednesday, May 8, 2023, at 8:30 am EDT in Baltimore.  The symposium will highlight development of gene therapy targeting oligodendrocytes for Canavan disease, updates on the company’s First in Human (FIH) open-label Phase 1/2 clinical trial using its proprietary vector (rAAV-Olig001-ASPA) and the discovery of novel oligodendrocyte targeting AAV capsids. Oligodendrocytes perform numerous key functions in the brain – including the production of myelin, the insulating material that enables proper neuronal function – and are now appreciated as having roles in a range of diseases. Myrtelle is developing a unique toolkit, including a proprietary class of recombinant adeno-associated virus (rAAV) vectors that directly target these cells thereby providing potential treatment options for diseases in the company pipeline.

Symposium speakers include:

  • Paola Leone, Ph.D., Professor and Director of the Cell and Gene Therapy Center at Rowan-Virtua SOM & School of Translational Biomedical Engineering & Sciences Virtua Health College of Medicine and Life Sciences of Rowan University will cover the history of her work in Canavan disease in her talk on the “Development of a Gene Therapy for Canavan Disease Targeting Oligodendrocytes.”
  • Rob Lober, M.D. Ph.D. Principal Investigator on Myrtelle’s Canavan Disease Gene Therapy Trial and Attending Neurosurgeon at Dayton Children’s Hospital and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine will present on a case study from Myrtelle’s Phase 1/2 First-in-Human Gene Therapy Clinical Trial in Patients with Canavan Disease in his talk titled, “Oligodendrocyte Targeted Gene Therapy for Canavan Disease.”
  • Patrick Aldrin-Kirk, Ph.D. Chief Science Officer at rAAVen, an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, will present on the “Discovery of Novel Oligodendrocyte Targeting AAV Capsids” and potential disease targets.

“As a leader in oligodendrocyte targeted therapies, we are thrilled to have the opportunity to host a symposium at ASGCT’s annual conference,” said Jordana Holovach, Head of Communications and Community at Myrtelle.  “This symposium provides a unique forum to highlight the critical but underappreciated role of oligodendrocytes in the overall health of the brain and as a potential target for treating neurodegenerative diseases. We are excited by the potential of our evolving toolbox for delivering therapeutic constructs to oligodendrocytes and impacting disease targets that are inherently intractable by nature.  We look forward to sharing our insights and generating further interest into the oligodendrocyte story at the symposium.”

Date: Wednesday, May 8, 2024

Location: Baltimore Convention Center – Room 324 – 326

Time: 8:30 am – 9:30 am


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:


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 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 under the identifier NCT04833907 or by emailing

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 results obtained to date continuing to reinforce the hypothesis that addressing the gene deficit directly in the brain, and specifically in oligodendrocytes, of patients with Canavan disease can potentially restore myelination and support functional improvements. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on 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 program demonstrating safety and efficacy, as well as future 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 clinical 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.

Media Contact:

Jordana Holovach
Head of Communications and Community
Myrtelle Inc.