The Babak Lab shared on LinkedIn:
“The FDA has approved the first therapy for glycogen storage disease type Ia (GSDIa).
Genglycos (pariglasgene brecaparvovec-opnr) received accelerated approval for patients aged 8 years and older. GSDIa is a rare genetic disease caused by glucose-6-phosphatase deficiency and has traditionally required strict, around-the-clock dietary management to prevent hypoglycemia.
Key Insights
- Genglycos is a one-time AAV8-based gene therapy that delivers a functional G6PC gene to the liver.
- In a randomized, placebo-controlled study, treatment reduced daily cornstarch intake by an average of 31% from baseline compared with placebo over 48 weeks.
- Patients also reduced their intake by approximately one cornstarch dose per day compared with placebo.
- The approval is accelerated, meaning additional trials are required to confirm clinical benefit.
Conclusion
This is the first approved treatment for GSDIa and an important step beyond dietary management toward addressing the underlying genetic cause of the disease. Long-term benefit and safety will continue to be evaluated.
A link to the FDA announcement.
Image generated using Sora by OpenAI.”
To which, Maria Babak, Associate Professor at City University of Hong Kong, added:
“One more gene therapy approved! Genglycos marks the 9th FDA-approved AAV gene therapy to date.”
You can also read ‘Cell and Gene Therapy 2026‘
