Search Results

Displaying Results 1-4 of 4 "'ASPA'"

May 11, 2026, 08:14 ET Myrtelle to Present Clinical Data and Regulatory Progress for MYR-101 Gene Therapy Program at ASGCT 2026

www.myrtellegtx.com.About Canavan DiseaseCanavan 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

More news about: Myrtelle, Inc


Oct 06, 2025, 08:18 ET Myrtelle Announces Presentation of Encouraging Interim Results from its Phase 1/2 Gene Therapy Trial for Canavan Disease at the 2025 Cell & Gene Meeting on the Mesa

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

More news about: Myrtelle, Inc


Sep 16, 2025, 08:11 ET Myrtelle Announces Nature Medicine Publication of Interim Results from Its Phase 1/2 Clinical Trial of Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease

rAAV-Olig001-ASPA (MYR-101) is the first-ever recombinant AAV gene therapy designed to target oligodendrocytes, the brain cells responsible for producing myelin. The therapy directly addresses the enzymatic deficiency caused by mutations in the ASPA gene, which leads

More news about: Myrtelle, Inc


Jul 08, 2025, 08:11 ET Myrtelle Launches Manufacturing of First-in-Class Gene Therapy for Canavan Disease in Strategic Alliance with Charles River and Viralgen

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

More news about: Myrtelle, Inc


Making a selection with these dropdown will cause content on this page to change. News search result will update as each option is selected.