Canavan disease (CD) is a rare genetic neurological disorder characterized by spongy degeneration of the white matter in the brain. Canavan is caused by mutations in the gene encoding the oligodendrocyte-specific enzyme aspartoacylase (ASPA), a critical enzyme for healthy white matter development and function. There currently is no cure for Canavan Disease, however research is being conducted in the scientific community to combat the disease and improve the lives of those who are diagnosed with it.
Myrtelle: How One Company is Changing Canavan Disease Treatment
Myrtelle is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating disorders of the central nervous system for which few, if any, treatment options exist. Myrtelle’s lead program is in Canavan Disease, a rare degenerative neurological disease first identified in 1931 by and named after Dr. Myrtelle Canavan, one of the first female neuropathologists.
Myrtelle is a patient-centered, innovation-driven, gene therapy company transforming the treatment of central nervous system disorders to benefit the livelihood of sufferers globally.
Myrtelle Gene Therapy: Innovative Research
Collaboration among highly skilled professionals is essential in gene therapy research, which is a slow and meticulous process involving careful planning, data analysis, and the utilization of cutting-edge biotechnology. Myrtelle is at the forefront of this field, building a scientific body of work that can be studied and utilized for future white matter disease solutions. A key aspect of Myrtelle’s research has been the advancement of their proprietary technologies to enhance the delivery of genes to myelin-generating cells, allowing for innovative approaches to address numerous disorders involving myelin.
A cornerstone of Myrtelle’s research has been advancing their proprietary technologies to better understand and optimize the delivery of genes to myelin-generating cells. This enables innovative approaches to address the needs of hundreds of disorders involving myelin. So how does this all work?
Myrtelle Clinical Trial: rAAV-Olig001-ASPA Gene Therapy
The ASPA gene is directly delivered to oligodendrocyte cells, responsible for myelin production, through Myrtelle’s proprietary vector, rAAV-Olig001-ASPA. Consequently, the oligodendrocytes can produce the Aspartoacylase (ASPA) enzyme. The ASPA gene is carried by an inactive virus that is delivered into the cerebrospinal fluid, which surrounds the brain and spinal cord, enabling the gene therapy to flow in and around the brain. Upon entering the cells, the ASPA gene may induce the production and release of ASPA, the enzyme required for the breakdown of N-Acetylaspartate (NAA). Essentially, the ASPA gene, once injected and inside the cells, may help the cells make and release ASPA, thereby reducing NAA levels and fighting Canavan disease, as suggested by researchers.
Clinical trials, including rAAV-Olig001-ASPA, are causing a stir in the white matter gene therapy field, providing scientists with invaluable insights and a glimpse into a more promising future. These trials have yielded a better comprehension of how this potential treatment functions and its potential effects on the brain. This information is instrumental in shaping a future white matter gene therapy landscape by offering scientists a novel treatment strategy.
Myrtelle: Taking The Fight To Canavan Disease
As stated before, currently there is no cure for Canavan disease. However this does not mean the scientific research community isn’t striving to improve the quality of life of those living with Canavan disease. Here are a few of the key proactive steps Myrtelle has been implementing to not only improve the lives of those affected but also potentially one day finding a cure:
- Utilizing Proprietary Technology
- Optimize the delivery of genes to myelin-generating cells.
- Innovative approaches to address the needs of hundreds of disorders involving myelin.
- Global patent families across a range of areas, including unique vector and gene sequences.
- Testing new routes of administration and methods of use in clinical trials.
- Cultivating Strategic Partnerships
- Forming a substantial group of strategic partners including: Pfizer, Rowan University, Dayton Children’s Hospital, amongst others.
- Scientific research, manufacturing, production and more, including an exclusive IP license with Pfizer Inc.
- Spreading Awareness
- Informative articles that tackle questions surrounding Myrtelle’s treatment approach.
- Social media posts to inform and educate the Canavan community and physicians on the trial including the manufacturing and regulatory process and updates on treated patients.
Myrtelle has been dedicated to educating the world about Canavan disease, committed to finding Canavan solutions and striving to be an innovative leader in the gene therapy space. Myrtell has been nothing short of a driving force in the fight against this devastating disease and is excited to help create a better future for all those affected. For more information and how you can get involved, visit Myrtelle’s website for the most up to date news surrounding the disease.
