RGX-111 Gene Therapy in Patients With MPS I

NCTID	NCT03580083 (View at clinicaltrials.gov)
Description	RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I. (Show More)
Development Status	Active
Indication	Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Disease Ontology Term	DOID:12802
Compound Name	RGX-111
Compound Description	AAV2/9.CB7.CMV.hIDUA.RBG pA
Sponsor	REGENXBIO Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	8
Results Posted	Not Available

Target Gene/Variant	IDUA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2/9
Editor Type
Dose 1	1E10 GC/g brain mass
Dose 2	5E10 GC/g brain mass
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States,Brazil,Israel

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	REGENXBIO is partnering with Nippon Shinyaku to continue clinical development