Myrtelle

The page covers news and developments in Myrtelle Inc’s gene therapy research.

Myrtelle develops and delivers novel treatments for devastating disorders of the central nervous system.

Myrtelle is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases.

Myrtelle publishes interim Phase 1/2 clinical trial results in *Nature Medicine* for its gene therapy **rAAV-Olig001-ASPA**, targeting **Canavan disease**. The trial evaluates safety and efficacy of the investigational therapy in patients. Findings appear in a peer-reviewed medical journal, marking progress in treating this rare neurological disorder.

Sep 2025Myrtelle, Inc.News article

Myrtelle begins commercial-scale production of its gene therapy for treating Canavan disease, a rare genetic disorder. The therapy represents the company’s first-in-class approach to addressing the condition. Manufacturing marks a key milestone in advancing the treatment toward patient access.

Jul 2025Myrtelle, Inc.News article

The FDA has chosen Myrtelle’s **rAAV-Olig001-ASPA**, a gene therapy candidate for **Canavan Disease**, for its **STAR pilot program**. The selection recognizes the therapy’s potential to address unmet medical needs in rare diseases and Myrtelle’s ability to expedite development toward market approval. The program supports clinical trials for CBER-regulated products with high clinical benefit.

Jun 2024Myrtelle, Inc.News article

Myrtelle reports **myelin and white matter increases** in patients treated with **rAAV-Olig001-ASPA gene therapy** in its first-in-human Canavan disease trial. Six-month MRI/MRS scans and validated assessments show **functional improvements** following treatment. The study evaluates the therapy’s potential to address neurological symptoms in affected patients.

Apr 2023Myrtelle, Inc.News article