Myrtelle Announces Presentation of Positive 12-month Post Treatment Data in its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy at the 2023 Cell & Gene Meeting on the Mesa held by the Alliance for Regenerative Medicine
Retrieved on:
Tuesday, October 10, 2023
Research, Neurology, Genetics, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Canavan disease, FIH, ASPA, White matter, Regenerative medicine, CD, Cell, Inc., Gene, Mutation, Tissue engineering, Clinical trial, Preyasi Raave, Gene editing, Natural history, Patient, Medical device
In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
Key Points:
- In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
- Following gene therapy, statistically significant gains in certain target white matter and myelin were observed.
- In turn, improvements in multiple domains on the validated Gross Motor Function Measure and Mullen Scales of Early Learning were demonstrated.
- Treated patients were routinely observed to outperform untreated age-matched natural history patients on the Mullen Scale of Early Learning.