Almac Clinical Services celebrates Grace Science, LLC in dosing first NGLY1 deficient patient with their innovative gene therapy

Almac Clinical Services is proud to celebrate Grace Science, LLC in reaching a critical milestone in their innovative GS-100 gene therapy programme for NGLY1 Deficiency— dosing their first patient in February 2024.

NGLY1 Deficiency is a life-threatening rare autosomal recessive disease with no approved therapy. The disease devastates the central nervous system, causing severe and debilitating symptoms, such as global developmental delay, cognitive impairment, movement disorders, and other neurological symptoms. Grace Science’s gene therapy, GS-100, is an AAV9 single-stranded viral vector that encodes a full-length version of the human NGLY1 gene.

Almac was honoured to support Grace Science in meeting this milestone, which required a fast-tracked dosing schedule to meet a momentous “dosing day” target in late February. The target date was especially significant as it coincided with Rare Disease Day.

Understanding that there was no room for error on the timeline, Almac’s clinical services teams from both the Souderton, PA and Durham, NC facilities sprang into action. Leading up to “dosing day,” the teams worked “all hands on deck” to ensure the high-value gene therapy product made it on time and in the right temperature sensitive condition to the site in Houston, Texas.

“I want to thank all of my colleagues across Almac that worked around the clock to dose patient 1 in Grace Science’s exciting new gene therapy trial,” said Todd Huml, Project Services Manager- Customer Support. “This was a true showing of departments across the company coming together to meet a patient’s need and truly Supply with Care.”

“This trial of a high-value, sub 80 degree gene therapy product has the potential to make a considerable impact on the lives of patients with NGLY1 Deficiency. Grace Science is a passionate partner and we are proud that Almac was able to play a role in this incredible milestone in their journey towards a cure.”