January 12, 2023
Read in 2 minutes
Myrtelle Inc. Announces Updated Results from an Open-Label Phase 1/2 Clinical Trial of Recombinant Adeno-Associated Virus Vector-Based Investigational Gene Therapy for Canavan Disease, a Deadly Genetic Disease of Children .
According to a release from Myrtelle, initial data from the first human study being conducted at Dayton Children’s Hospital in Ohio showed that all eight patients had 3-monthly follow-up visits. In addition, most patients showed a reduction in cerebrospinal fluid volume evidenced by MRI.
Positive interim data from a Phase 1/2 clinical trial of a novel gene therapy to treat Canavan disease have been presented. Source: Adobe Stock
In addition, according to this release, clinical measures of motor and cognitive function using the Mullen Scale of Gross Motor Function and Early Learning Tools are consistent with the continued clinical decline expected in patients with untreated Canavan disease. In contrast, provided improvements in several areas.
“As we continue to follow patients and their progress, we are encouraged by the early findings of Myrtelle’s Phase 1/2 clinical trial in Canavan disease.” mark picket,doctorate, Myrtelle’s CEO told Healio: “We look forward to meeting with regulatory authorities for expedited approval of this gene therapy to provide it to patients who currently have no treatment options.”
The first 3 patients in the first cohort to receive new therapy were at least 18 months old from the start of treatment. Evaluation of these initial participants at later time points also showed improvements in imaging and validated functional scale measures. No serious drug-related adverse events have been observed in treated patients to date, the company said in a release.
The current study utilizes Myrtelle’s proprietary recombinant adeno-associated virus vector to directly target oligodendrocytes, Canavan disease-affected brain cells involved in the production of myelin. This oligodendrocyte-targeted gene therapy restores the function of the deficient aspartoacylase enzyme in patients with Canavan disease, promoting her N-acetylaspartate metabolism and brain development in these patients. is intended for
According to the release, the investigational gene therapy has received Orphan Drug, Rare Pediatric and Fast Track designations from the FDA, as well as Orphan Drug designation and Advanced Therapeutic Classification from the European Medicines Agency. I’m here.
“The early results so far from patients treated in the Phase 1/2 trial of Myrtelle are encouraging.” Rob roverMD, Principal investigator and neurosurgeon attending Dayton Children’s Hospital said in a release: “Given these data, we encourage continued follow-up of patients receiving these treatments and further development of gene therapy candidates to bring this potential treatment option to patients. [Canavan disease] Patience. “
