Ultragenyx Announces the Publication of a Successful 96-Week Randomized, Placebo-Controlled Trial with Crossover Treatment of GENGLYCOS™ (also known as DTX401) AAV Gene Therapy in GSDIa in The Journal of Inherited Metabolic Disease
Tuesday, September 1, 2026 BiopharmaWatch Research 1 min read
Key Takeaway: Ultragenyx has published results from a 96-week randomized, placebo-controlled trial of its AAV gene therapy, GENGLYCOS™ (DTX401), for GSDIa. The trial showed a mean reduction of 61% in daily cornstarch intake among participants, with many maintaining glycemic control. Notably, one-third of participants completely eliminated nighttime cornstarch dosing.
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POSITIVE FACTORS
Significant reduction in daily cornstarch intake by 61%.
Most participants maintained glycemic control.
33% of participants eliminated nighttime cornstarch dosing.
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Full Press Release Details
At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50% Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401
Frequently Asked Questions
What were the results of the DTX401 trial?
The trial showed a 61% reduction in daily cornstarch intake while maintaining glycemic control.
How many participants eliminated nighttime dosing?
33% of participants completely eliminated nighttime cornstarch dosing.
What is GENGLYCOS™ used for?
GENGLYCOS™ (DTX401) is an AAV gene therapy for treating GSDIa.
Where were the trial results published?
The results were published in The Journal of Inherited Metabolic Disease.