Canavan Disease Gene Therapy Continues to Show Promising Data


Myrtelle’s rAAV-Olig001-ASPA was granted fast track, rare pediatric disease, and orphan drug designations in March 2022.

Myrtelle’s gene therapy rAAV-Olig001-ASPA has shown efficacy and safety in patients with Canavan disease, with improvements in white matter and myelin, according to updated data from a phase 1/2 trial (NCT04833907).1

These data were presented by Armen Asatryan, MD, chief medical officer, Myrtelle, at the National Tay Sachs and Allied Diseases Annual Family Conference, July 7-10, held in Denver, Colorado. The new data include 6-month post treatment analyses of the first 3 patients that demonstrated increases of white matter and myelin in the brain as measured by MRI. These patients also experienced improvements on the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL) from baseline. These data contrast with the natural progression of continuous deterioration with Canavan disease.

“The 6-month functional and anatomic data observed in the first three patients elicit hope when we compare it with the natural course of this devastating disease. Equally encouraging are positive changes in the patients’ reflexes, eye tracking, vocalizations to communicate, and overall awareness” co-principal investigator Robert Lober, MD, PhD, FAANS, associate professor of pediatrics, Wright State University Boonshoft School of Medicine and Attending Neurosurgeon, Dayton Children’s Hospital, said in a statement.1 “These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.”

The phase 1/2, first-in-human trial is evaluating rAAV-Olig001-ASPA, Myrtelle’s proprietary vector designed to directly target oligodendrocytes. The gene therapy then restores aspartoacylase (ASPA) function and enables N-Acetylaspartate (NAA) metabolism to support functional myelin production prevented by a mutation in theASPA gene.

READ MORE: Gene Therapy for Canavan Disease Shows Early Promise in Safety and Pharmacodynamics

All patients in the trial receive 3.7 x 1013 vg delivered by intracerebroventricular (ICV) injection of the gene therapy. The trial has treated 5 patients so far and investigators have observed no treatment-related adverse events and a favorable safety and tolerability profile.

“Myrtelle’s current investigational gene therapy targeting oligodendrocytes with a unique rAAV vector builds upon over two decades of science and effort with the potential, if successful, to usher in new treatment options for children with Canavan disease,” Asatryan added to the statement.1 “We intend to build on these encouraging initial findings and complete our current Phase 1/2 clinical study, following which we plan to engage with regulatory authorities to advance the program to the later stages of clinical development.”

The trial is being conducted at Dayton Children’s Hospital in Ohio. It is currently recruiting, with an expected enrollment of 24 participants with additional patients enrolled in the second quarter of 2022.2 In March 2022, the FDA granted fast track, rare pediatric disease, and orphan drug designations to rAAV-Olig001-ASPA for Canavan disease.3

1. 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. News release. Myrtelle. July 8, 2022.
2. Myrtelle announces positive preliminary clinical data for its proprietary gene therapy in canavan disease. News release. Myrtelle. February 15, 2022.
3. Myrtelle Receives FDA Fast Track, Rare Pediatric Disease, and Orphan Drug Designations for its Proprietary Gene Therapy for the Treatment of Canavan Disease. News release. Myrtelle. March 15, 2022.
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