Gene Therapy Study for Children With CLN5 Batten Disease

NCTID	NCT05228145 (View at clinicaltrials.gov)
Description	This is a prospective, non-randomized, open-label, dose escalation study of a single administration of gene therapy in children who are 3 to 9 years old with Neuronal Ceroid Lipofuscinosis (Batten) Subtype 5 (CLN5) disease. (Show More)
Indication	Neuronal Ceroid Lipofuscinosis CLN5
Compound Name	NGN-101
Sponsor	Neurogene Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	6

Target Gene/Variant	CLN5
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 intermediate dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States,United Kingdom

Has US IND	True
Recent Updates	RMAT application was denied by FDA, company discontinued the program