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Expert Interview

Slingshot members are talking to an expert! The topic is:

Examining the potential of Ultragenyx's DTX401 Phase 3 Trial Results in treating Glycogen Storage Disease Type Ia (GSDIa)

Ticker(s): RARE

Who's the expert?

Institution: Self employed

  • Pediatric Endocrinologist & Consultant for rare disease trials and former Director of the Glycogen Storage Disease Program at Boston Children's.
  • Led the submission of the IND for gene therapy for NGLY1 deficiency, and remains a consultant for rare disease trials.
  • Served as the medical lead or the primary consultant for 7 successful IND submissions.

Interview Questions
Q1.

The Phase 3 GlucoGene study reported a statistically significant reduction in daily cornstarch intake with DTX401. Can you explain the clinical importance of this reduction for GSDIa patients?

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Q2.

One of the key outcomes was the maintenance of glucose control while reducing cornstarch intake. How does DTX401 achieve this balance, and what does it mean for the daily management of GSDIa?

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Q3.

The study also met secondary endpoints, including a reduction in the number of daily cornstarch doses. How might these reductions affect the quality of life for patients and their families?

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Q4.

DTX401 demonstrated an acceptable safety profile with manageable hepatic effects. What are the main safety concerns with gene therapy in GSDIa, and how were they addressed in this study?

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Q5.

The Patient Global Impression of Change (PGIC) showed varying degrees of improvement. How do these subjective measures correlate with the objective findings, and what do they tell us about the patient experience?

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Q6.

Given the durable response observed in the Phase 1/2 study with reductions in cornstarch intake lasting up to 5 years, what are the long-term implications of DTX401 treatment for GSDIa patients?

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Q7.

With plans to discuss these Phase 3 results with regulatory authorities to support a marketing application, what are the key milestones and challenges in bringing DTX401 to market?

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Q8.

Ultragenyx is committed to following patients for at least 10 years post-DTX401 infusion. What additional data do you hope to gather from this long-term follow-up, and how might it influence future research and treatment strategies for GSDIa and other similar conditions?

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