Myrtelle has successfully treated three children from Cohort 1 of the first-in-human Phase I/II clinical trial of gene therapy for Canavan disease (CD).

So far, three CD patients aged three to five years in Cohort 1 received the treatment at Dayton Children’s Hospital in the US.

The Data Monitoring Committee (DMC) analysed the data from the Cohort 1 patients and advised expansion of the trial to two younger cohorts: patients aged 15-36 months and below 15 months.

The CD trial leverages a recombinant adeno-associated virus (rAAV) vector that facilitates targeting of oligodendrocytes, which are vital for myelination and development of the brain.

Oligodendrocytes cells are present in the brain and responsible for producing an insulating material called myelin that enables neurons to function properly. 

CD is a fatal childhood genetic brain disorder in which aspartoacylase gene (ASPA) mutations hinder the normal expression of Aspartoacylase.

The therapy aims to reinstate the functioning of ASPA, thereby aiding in the metabolism of N-acetylaspartic acid (NAA) and myelination. 

A vital enzyme, aspartoacylase is produced in oligodendrocytes that disintegrates the neurochemical NAA.

NAA collects in the brain and hinders the normal bioenergetics, myelin creation, and brain health if not broken down by oligodendrocytes.

Reduced head control, eye tracking difficulty, a larger head size, increased irritability, reduced muscle tone and delays in attaining motor milestones typical are symptoms of CD.

Last year, Myrtelle and Pfizer signed an exclusive global licensing agreement for the development and marketing of gene therapy to treat CD.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

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