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)
Development Status	Active
Indication	NGLY1 Deficiency
Disease Ontology Term	DOID:0060728
Compound Name	GS-100
Sponsor	Grace Science, LLC
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	6 (ESTIMATED)
Results Posted	Not Available

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	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed medium dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation, Support for Clinical Trials Advancing Rare Disease Therapeutics
Recent Updates	Selected for START program (6/3/24)