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
In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
- 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.