Preyasi Raave

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

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.

Myrtelle Announces Presentation at Hydrocephalus Association & Rudi Schulte Research Workshop on Developing Non-Invasive Hydrocephalus Therapies: Molecular and Cellular Targets

Retrieved on: 
Thursday, September 28, 2023

These improvements in treated patients contrast the deterioration in untreated age-matched CD patients based on natural history.

Key Points: 
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients based on natural history.
  • MRI-based improvements are correlated with improvements in motor and cognitive function in treated patients, which will be presented at upcoming conferences.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and brain development in patients with CD.

Myrtelle to Present Positive 6-month Post-Treatment Data in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay-Sachs and Allied Diseases Annual Family Conference

Retrieved on: 
Wednesday, May 31, 2023

Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.

Key Points: 
  • Olga Flamini, MD, PhD, Medical Director at Myrtelle, will deliver a presentation on Friday, June 2, 2023.
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients in Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.
  • “Connecting with patients and caregivers allows us to incorporate the patient voice into our drug development activities.”

Myrtelle Announces Presentation of Positive 6-month Post-Treatment Data in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the American Society of Gene and Cell Therapy 26th Annual Meeting

Retrieved on: 
Tuesday, May 16, 2023

Clinical measurements of motor and cognitive function using validated assessment scales demonstrate mean absolute and percent improvements across multiple domains.

Key Points: 
  • Clinical measurements of motor and cognitive function using validated assessment scales demonstrate mean absolute and percent improvements across multiple domains.
  • Improvements in multiple anatomic and biomarker measurements by magnetic resonance imaging (MRI) and spectroscopy (MRS) have also been observed.
  • These improvements in treated patients contrast the deterioration in untreated age-matched CD patients in Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme aspartoacylase.

Myrtelle Announces Positive 6-month Post-Treatment Data in Patients in Its First-in-Human Clinical Study of rAAV-Olig001-ASPA Gene Therapy in Canavan Disease

Retrieved on: 
Wednesday, April 19, 2023

In several domains, the observed improvement in treated patients are in contrast to the observed deterioration in untreated age-matched CD patients within Myrtelle’s natural history data set.

Key Points: 
  • In several domains, the observed improvement in treated patients are in contrast to the observed deterioration in untreated age-matched CD patients within Myrtelle’s natural history data set.
  • In CD, normal brain development is impaired due to a mutation in the ASPA gene that encodes the enzyme Aspartoacylase (ASPA).
  • The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function and hence the metabolism of NAA and brain development in patients with CD.
  • In addition, volumetric MRI (magnetic resonance imaging) measurements showed increases in multiple brain tissue compartments and reductions in CSF volume.