Category: Gene Therapy

Gene Therapy & Disease Burden Impact

The Oligodendrocyte Advantage

For decades, central nervous system (CNS) gene therapy has approached the brain as single, undifferentiated target. Myrtelle was built on a different premise: the cell type matters as much as the delivery route.

Beyond the Biomarkers

As we work to advance gene therapy for rare, fatal genetic conditions such as Canavan disease, it is easy to become focused on the data points and statistics that are essential for regulatory progress. Yet those numbers tell only part of the story.

Proven Durability: Groundbreaking Gene Therapy Persistence Data

Groundbreaking data has offered new insights into Canavan disease an ultra rare and fatal genetic brain disorder.

Real-World Signals from Treated Children

Clinical trials are signaling fascinating insights into Canavan diease, an ultra rare and fatal genetic brain disorder.

Exciting Update! Explore Myrtelle’s Insightful Slide Presentation on Gene Therapy for Canavan Disease Preliminary One-Year Results from Meeting on the Mesa

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’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.

Our Approach Has The Potential To Correct Myelin-Based Deficits In A Range Of Disorders

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 Enters into a Worldwide Exclusive License Agreement with Rescue Hearing to Develop and Commercialize Gene Therapy for the Treatment of Hearing Loss

Myrtelle Inc. has entered into a worldwide exclusive licensing agreement with Rescue Hearing Inc. to develop a novel gene therapy for DFNB8 genetic hearing loss, aiming to repair and restore damaged cells and promote neuron survival, offering potential hope for patients with this condition.

Myrtelle Inc. Announces Expansion of Its Pipeline with a Novel Gene Therapy Program for Pelizaeus-Merzbacher Disease (PMD)

Myrtelle Inc. announced the launch of a new research program for Pelizaeus-Merzbacher Disease (PMD), a rare brain disorder, using gene therapy to target oligodendrocytes and address the underlying cause of myelin production disruption.

Health Care Hero: Dayton Children’s doctor offers hope, groundbreaking clinical trials for patients

Dr. Robert Lober is a leading pediatric neurosurgeon and brain cancer researcher at Dayton Children’s Hospital.