NEW YORK, May 7, 2024 ~ Myrtelle Inc., a clinical-stage gene therapy company, will be hosting a symposium at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting on Wednesday, May 8, 2023. The symposium will focus on Canavan disease (CD) clinical trial updates and future directions for oligodendrocyte targeting AAVs.

Canavan disease is a fatal childhood genetic brain disease caused by mutations in the ASPA gene. This prevents the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. These cells play a crucial role in brain function by producing myelin, the insulating material that enables proper neuronal function.

The symposium will feature speakers such as 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. She will discuss the history of her work in Canavan disease and the development of a gene therapy targeting oligodendrocytes.

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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 a case study from Myrtelle's Phase 1/2 First-in-Human Gene Therapy Clinical Trial in Patients with Canavan Disease. His talk is 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 gene therapies, will also be presenting. He will discuss the discovery of novel oligodendrocyte targeting AAV capsids and potential disease targets.

Jordana Holovach, Head of Communications and Community at Myrtelle, expressed excitement about the symposium, stating that it provides a unique opportunity to highlight the critical role of oligodendrocytes in brain health and their potential as a target for treating neurodegenerative diseases. She also mentioned the company's evolving toolbox for delivering therapeutic constructs to these cells and its potential impact on previously untreatable diseases.

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The symposium will take place at the Baltimore Convention Center in Room 324-326 from 8:30 am to 9:30 am. Myrtelle Inc. is a gene therapy company focused on developing treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches.

Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. More information on Myrtelle's clinical trial in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing [insert email address]. For more information about Myrtelle, please visit their website at www.myrtellegtx.com.

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