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Promising initial results in gene therapy trial

From a press release dated 4 January 2019

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive topline safety and efficacy data from the first, lowest dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSD1a). A biologic response, reflected by improved glucose control and increased time to hypoglycemia during fasting, was observed in all three patients, with two patients demonstrating a clinically meaningful improvement in time to hypoglycemia during a controlled fasting challenge.

“Two of three patients in this first dose cohort experienced a sufficient increase in time to hypoglycemia such that they may be able to sleep through the night without taking supplemental cornstarch,”
said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit.

“In addition to the promising results observed with the controlled fasting challenge, all three patients have demonstrated improvement in glucose control throughout the day, and all three patients have been able to decrease their daily cornstarch intake by approximately half. We are extremely encouraged by these results and look forward to beginning enrollment in the second cohort this month, with results expected in mid-2019.

“We are very pleased with what we are seeing in this first cohort of patients. After 20 years of research in preparation for this study, it is extremely rewarding to see such positive results on the low dose. We are excited about the potential as the dose is increased,”

said David Weinstein, M.D., M.M.Sc., Professor and Director, Glycogen Storage Disease Program at Connecticut Children’s Medical Center and UConn Health.

Go to the full press release.