A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy with TTX-381 for the Ocular Manifestations Associated with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

NCTID	NCT05791864 (View at clinicaltrials.gov)
Description	This is a first-in-human, open-label, single ascending dose study of TTX-381 for the treatment of ocular manifestations of CLN2 (Batten disease). (Show More)
Development Status	Active
Indication	Neuronal Ceroid Lipofuscinosis Type 2
Disease Ontology Term	DOID:0110726
Compound Name	TTX-381
Compound Alias	RGX-381
Compound Description	AAV9.CB7.hCLN2
Sponsor	Tern Therapeutics, LLC
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	16 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	TPP1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal, intracisternal (RGX-181)
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	2E10 gc/eye
Dose 2	6E10 gc/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United Kingdom

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Tern Therapeutics acquired RGX-381 and RGX-181 from REGENXBIO August 2024; Dose escalation cohort is fully enrolled, enrollment in expansion cohort is ongoing