Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

NCTID	NCT06199531 (View at clinicaltrials.gov)
Description	A non-randomized, open-label, dose escalation study of a single intracerebroventricular (ICV) administration of a gene replacement therapy in subjects who are 2 to 18 years old with NGLY1 Deficiency. (Show More)
Indication	NGLY1 Deficiency
Compound Name	GS-100
Sponsor	Grace Science, LLC
Funder Type	Industry
Status	Recruiting
Enrollment Count	12

Target Gene/Variant	NGLY1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracerebroventricular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	Undisclosed low dose
Dose 2	Undisclosed medium dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	Selected for START program (6/3/24)