- Jordana Holovach (04.23.2024) 3 mins
- (Gene Therapy)
Phase 1/2 Clinical Trial: Paving a Path of Hope for Canavan Disease
Canavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreCanavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreCanavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreCanavan disease is a complex ailment and a member of rare diseases discovered in the 20th century.
Read MoreRecent advancements in rare disease technology have catapulted gene therapy research to new heights.
Read MoreA Q&A with Rick Layer, Vice President, Myrtelle’s Head of Pharmacology and Translational Science
Read MoreCheryl Demos Fludas, Head of Clinical Operations at Myrtelle, explains the process of enrolling a patient in an experimental clinical trial, emphasizing the importance of informed consent and eligibility criteria.
Read MoreChristopher J. Morrison, Senior Vice President of Technical Operations, discusses the complexities of gene therapy manufacturing, the importance of cGMP standards, manufacturing timelines and challenges, yield determination, and the stringent measures taken to ensure the safety and quality of gene therapy products.
Read MoreIn this Q&A with Richard Layer, Vice President and Head of Pharmacology, key insights into the central function of oligodendrocytes and myelin generation, the role of Myrtelle’s novel class of nonpathogenic rAAV vectors in gene therapy for myelin-related disorders, and the expansion of Myrtelle’s pipeline to address Pelizaeus-Merzbacher Disease (PMD) are discussed. Additionally, the evolving understanding of oligodendrocytes’ importance in the central nervous system and their potential implications for various neurological and psychiatric disorders is highlighted.
Read MoreNancy Barone Kribbs, Senior Vice President of Global Regulatory Affairs at Myrtelle, discusses the regulatory path for Myrtelle’s rAAV-Olig001-ASPA gene therapy, emphasizing the importance of establishing efficacy and safety in drug development for treating Canavan disease patients.
Read MoreArmen’s passion is to bring novel transformational treatments to rare disease patients through innovative modalities.
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