Biopharmaceutical company Ultragenyx Pharmaceutical has announced dosing of the first patient in the Phase 1/2 study of DTX401, an adeno-associated virus vector-based gene therapy for the treatment of patients suffering with glycogen storage disease type Ia (GSDIa).

Connecticut Children’s Medical Center and UConn Health Glycogen Storage Disease Program director David Weinstein said: “For over 20 years, our team and the team at the National Institutes of Health (NIH) have worked for this day to occur. We are thrilled to see the gene therapy trial for GSD type Ia beginning.

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“I am confident that patients with this condition around the world could benefit from this trial along with children and adults with other genetic liver diseases. This is truly an exciting day for the GSD community.”

The open-label Phase 1/2 study assesses the safety, tolerability and therapeutic response of DTX401 in adults with GSDIa.

Important efficacy assessments of the study consist of time to hypoglycemia, impact on biomarkers such as lipids, uric acid, and measurement of glycogen in liver.

“For over 20 years, our team and the team at the National Institutes of Health (NIH) have worked for this day to occur.”

The study involves three potential dosing cohorts, and three patients will be enrolled in each cohort. Patients in the first cohort receive a single dose of DTX401 of 2.0 × 10^12 GC/kg.

Once the data monitoring committee assesses the safety data for all patients in the first dosing cohort, the decision to proceed to a higher-dose cohort will be made.

GSDIa is a genetically inherited glycogen storage disease caused by a defective gene for the enzyme glucose-6-phosphatase-α (G6Pase-α), which leads to the body’s inability to regulate blood sugar (glucose).

DTX401 is an investigational adeno-associated virus vector (AAV) type 8 gene therapy that delivers stable expression and activity of G6Pase-α after a single intravenous infusion.

Data from the three-patient first-dose group is expected in the second half of this year.

Set up in 2010, Ultragenyx Pharmaceutical has built a portfolio of approved therapies and product candidates aimed at diseases with high unmet medical need, for which there are no approved therapies.