Myrtelle, via Business Wire News, reports the completion of Phase 1/2 investigating its gene therapy for Canavan disease (CD) which they have now expanded to treat younger patients. Thus far, three young children aged three to five have received treatment.
Upon reviewing the children’s data, a decision was made to open the trial to two younger groups: ages fifteen to thirty-six months and under fifteen months.
About Canavan Disease
Canavan Disease is a rare, fatal childhood disease that causes the white matter in the brain to deteriorate. CD is a genetic disorder. Myrtelle’s gene therapy utilizes a modified (recombinant) adeno-associated virus (rAAV) vector that enables targeting of central nervous system cells (oligodendrocytes). The central nervous system cells (CNS) are critical as they produce an insulating layer called myelin that protects nerves in the spinal cord and brain. This permits electrical impulses to travel efficiently along nerve cells.
In CD, an ASPA gene mutation affects the production of myelin. The ASPA gene encodes for the hydrolase enzyme Aspartoacylase
Myrtelle’s gene therapy seeks to restore the function of ASPA to enable the metabolism of N- acetylaspartic acid, a chemical that supports myelination.
Canavan disease affects the muscles, central nervous system, eyes, and causes seizures. More information about Canavan disease is available here.
A History of Gene Therapy to Treat CD
In 2021 Myrtelle and Pfizer, Inc. signed a global licensing agreement to develop gene therapy to treat CD.
In 1998, the FDA approved the first clinical trial investigating the use of a liposomal vector in gene therapy. The trial was conducted by Professor Paola Leone, Director, who currently conducts gene and cell therapy for Rowan University.
Dr. Leone’s work, as well as that of his associates, was instrumental in leading to the first application of AAV vectors in CD. The First-in-Human international trial began in 2000 and used Adenno-Associated Virus Type 2 Wild Type.
Over the next few decades, the doctor and his associates continued their progress. This eventually allowed Myrtelle to enter the field with its ASPA gene therapy for CD patients.
Dr. Leone recently commented on the progress being made in gene therapy through clinical trials. He stated further that it has been his life-long mission to provide advances for children with CD.
Just as in the past, Myrtelle Inc. continues to focus on technologies and programs in gene therapy to treat neurological diseases.
