Myrtelle to Host Symposium on Canavan Disease Clinical Trials and Future Oligodendrocyte AAVs at ASGCT Meeting in Baltimore, May 8, 2024
NEW YORK, May 7, 2024 -- Myrtelle Inc., a clinical-stage gene therapy company committed to creating transformative treatments for neurodegenerative diseases, is set to host a symposium focusing on Canavan disease (CD) clinical trial updates and future directions for oligodendrocyte-targeting AAVs. This symposium will take place at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting on May 8, 2023, at 8:30 am EDT in Baltimore.
The event will spotlight the development of gene therapy targeting oligodendrocytes for Canavan disease, provide updates on the company's First in Human (FIH) open-label Phase 1/2 clinical trial utilizing its proprietary vector (rAAV-Olig001-ASPA), and discuss the discovery of new oligodendrocyte-targeting AAV capsids. Oligodendrocytes are crucial cells in the brain responsible for producing myelin, an insulating material vital for proper neuronal function. They have been increasingly recognized for their roles in various diseases. Myrtelle is working on a unique set of tools, including a proprietary class of recombinant adeno-associated virus (rAAV) vectors that directly target these cells, potentially offering treatment options for several diseases in the company's development pipeline.
Speakers at the symposium include:
- Dr. Paola Leone, 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. Dr. Leone will discuss the history and development of gene therapy for Canavan disease targeting oligodendrocytes.
- Dr. Rob Lober, Principal Investigator on Myrtelle's Canavan Disease Gene Therapy Trial and Attending Neurosurgeon at Dayton Children's Hospital, who is also an Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine. Dr. Lober will present a case study from Myrtelle's Phase 1/2 First-in-Human Gene Therapy Clinical Trial in Patients with Canavan Disease.
- Dr. Patrick Aldrin-Kirk, Chief Science Officer at rAAVen, an AAV engineering company focusing on creating innovative vectors for the next generation of gene therapies, will cover the discovery of novel oligodendrocyte-targeting AAV capsids and their potential disease targets.
Jordana Holovach, Head of Communications and Community at Myrtelle, expressed excitement about hosting the symposium. She highlighted the significance of oligodendrocytes in maintaining brain health and their potential as targets for treating neurodegenerative diseases. Holovach emphasized the potential impact of Myrtelle's evolving toolbox for delivering therapeutic constructs to oligodendrocytes and addressing challenging disease targets. She looked forward to sharing insights and generating further interest in the role of oligodendrocytes at the symposium.
Event details are as follows:
Date: Wednesday, May 8, 2024
Location: Baltimore Convention Center – Room 324 – 326
Time: 8:30 am – 9:30 am
Myrtelle Inc. is a pioneering gene therapy company dedicated to developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and a portfolio of programs and technologies that support innovative gene therapy approaches. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program.
Canavan disease (CD) is a fatal genetic brain disorder in children caused by mutations in the ASPA gene, which inhibits the normal production of the enzyme aspartoacylase by oligodendrocytes. This deficiency hampers brain development and myelin production. Symptoms generally appear within the first few months of life, including poor head control, large head size, difficulty tracking with the eyes, irritability, low muscle tone, and delays in motor milestones. As the disease progresses, it leads to seizures, spasticity, swallowing difficulties, and severe muscle deterioration, with most children facing life-threatening complications by age 10. Currently, there is no cure for Canavan disease, and only palliative treatments are available.
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