Category: Canavan Disease

Myrtelle to Present Breakthrough Insights on FDA’s Groundbreaking START Pilot Program at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting in New Orleans on Thursday, May 15th

Myrtelle Announces Significant Reduction in N-Acetylaspartate (NAA), a Key Biomarker, in Patients Treated in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease 

Inheritance Patterns of Canavan Disease: Navigating Options When Only One Mutation is Identified

Myrtelle Announces Presentation of Positive 12-month Post Treatment Data in its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy at the 2023 Cell & Gene Meeting on the Mesa held by the Alliance for Regenerative Medicine

Myrtelle Announces Positive 6-month Post-Treatment Data in Patients in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease

Analyses at 6 months showed increases in myelin, white matter and other MRI and MRS measurements as well as functional improvements on validated assessment scales Encouraging initial efficacy data support further development of rAAV-Olig001-ASPA as a potential therapeutic approach for children with Canavan disease

Gene Therapy – Measuring its Impact with Dr. Christopher Janson

Dr. Christopher Janson, the Principal Investigator on Myrtelle’s Canavan Disease gene therapy trial, addresses questions frequently asked by the families in relation to the disease and gene therapy

Myrtelle Completes Dosing of 8 Patients with Canavan Disease in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA

Myrtelle Inc. has successfully completed dosing 8 patients with Canavan Disease in its Phase 1/2 clinical trial for the investigational gene therapy rAAV-Olig001-ASPA, demonstrating favorable safety and tolerability, along with significant improvements in functional scales and brain white matter content in early assessments.

Myrtelle’s rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Advanced Therapy Medicinal Product Classification from the European Medicines Agency

Myrtelle Inc. has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA) for its lead gene therapy product candidate, rAAV-Olig001-ASPA, aimed at treating Canavan disease, facilitating its regulatory pathway and development in the EU.

Myrtelle Announces Positive Data for Its investigational Proprietary rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay Sachs & Allied Diseases Association Conference

Analyses of the first three patients at 6 months post-treatment showed increases in white matter and myelin and improvements on validated functional scales To date, 5 patients have been treated in the ongoing Phase 1/2 First-in-Human clinical trial with favorable safety and tolerability

Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease

Myrtelle Inc., a gene therapy company, has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations for its gene therapy aimed at treating Canavan Disease, highlighting the urgency of developing a treatment for this devastating childhood disease.

Myrtelle Announces Positive Preliminary Clinical Data for Its Proprietary Gene Therapy in Canavan Disease

Myrtelle’s Phase 1/2 clinical trial introduces first-of-its-kind proprietary recombinant adeno-associated virus (rAAV) vector designed to enable targeting of oligodendrocytes.

Myrtelle Announces Successful Completion of Initial Stage of Phase 1/2 Clinical Trial of Proprietary Gene Therapy for Canavan Disease and Expands Treatment to Younger Patients

Myrtelle Inc. has announced the successful administration of gene therapy in its Phase 1/2 clinical trial for Canavan disease, treating three children aged 3 to 5 years, with plans to expand the study to younger age groups based on recommendations from the Data Monitoring Committee.

Myrtelle Announces Exclusive Worldwide Gene Therapy Licensing Agreement for Canavan Disease

Myrtelle Inc. has entered into an exclusive worldwide licensing agreement with Pfizer Inc. for an investigational recombinant adenovirus (rAAV) gene therapy aimed at addressing Canavan disease (CD), a devastating childhood genetic neurological disorder.