A new treatment for Rett syndrome is officially being studied in humans in Canada, meaning Canadian patients may have a new, viable treatment option in the future. Called TSHA-102, this gene therapy will move forward in clinical trials after demonstrating positive data in mice models of this rare neurological disorder. Want to learn more? You can find the source article at Neuro Central.
About Rett Syndrome
First up, we should know what Rett syndrome is. Rett syndrome is a severe, rare neurological disorder that primarily affects females. It was once thought to be a form of autism, but has since been distinguished as its own condition. It is a genetic disorder, with a mutation occurring on the X chromosome. The exact location of this mutation and its effects are unknown, but researchers do know that it is typically a sporadic mutation. Symptoms of this condition usually appear between the first 12-18 months of life. Effects include slowed brain growth, a small head, issues with muscle coordination, social anxiety, lack of language skills, seizures, uncoordinated breathing, and a tense or irritable disposition.
After these symptoms are noticed, doctors will conduct a clinical examination and rule out other conditions, such as autism. Genetic testing will be used to confirm, and it can also show the severity of a specific case. There is no cure for Rett syndrome, and treatment consists of symptom management. Doctors will suggest physical, speech, and occupational therapy. They may also prescribe anti-seizure medications.
Development of TSHA-102
The approval of the Clinical Trial Application (CTA) to Health Canada hinged upon positive data from preclinical studies. Both mouse and non-human primate (NHP) models of Rett syndrome were utilized to investigate TSHA-102, both of which demonstrated favorable results. The mouse models saw improvements in survival, alongside motor and respiratory functions. On the other hand, NHP models demonstrated the bio distribution of the gene therapy, proving its mechanism of action. These models also experienced the favorable safety and tolerability profile of the drug.
Armed with these data, Taysha Gene Therapies – the developer of TSHA-102 – submitted a CTA to Health Canada, which approved the application. Now, the gene therapy company can move forward with its Phase 1/2 trial in Montreal’s Sainte-Justine Mother and Child University Hospital Center. Here, it will investigate TSHA-102, which utilizes Taysha’s novel miRARE platform.
Initial data from this clinical trial is expected to be released by the end of the year. Hopefully, these results will be just as promising as preclinical data, as it would suggest a new treatment option for Rett syndrome patients. In fact, if TSHA-102 is approved, it will become the first gene therapy available to this patient population.