Orotic acid

Ultragenyx Announces Positive Multi-Year Durability Data from Ongoing Phase 1/2 Gene Therapy Studies and Data on HeLa 3.0 Manufacturing Platform at American Society of Gene & Cell Therapy 2021 Annual Meeting

Retrieved on: 
Friday, May 14, 2021

The primary endpoint is expected to be the reduction in oral glucose replacement with cornstarch while maintaining or improving glucose control assessed by continuous glucose monitoring.

Key Points: 
  • The primary endpoint is expected to be the reduction in oral glucose replacement with cornstarch while maintaining or improving glucose control assessed by continuous glucose monitoring.
  • These cell line engineering improvements result in a 2- to 5-fold yield improvement over the HeLa 2.0 platform, and a 50-fold yield improvement over HeLa 1.0.
  • It is caused by a defective gene coding for the enzyme G6Pase-\xce\xb1, resulting in the inability to regulate blood sugar (glucose).
  • It has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism.