Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Phase 1/2 Completed 4 enrolled UniQure Biopharma B.V. Institut Pasteur · collab Venn Life Sciences · collab

NaSingle-groupOpen-labelTreatment

Summary

This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.

Timeline

Start: 2013-09-17
Primary completion: 2019-11-27
Completion: 2019-11-27

Publications

Gougeon ML, Poirier-Beaudouin B, Ausseil J, Zerah M, Artaud C, Heard JM, Deiva K, Tardieu M. Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome. Front Immunol. 2021 May 10;12:655478. doi: 10.3389/fimmu.2021.655478. eCollection 2021.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	rAAV2/5-hNAGLU	Gene therapy (AAV / viral vector)	4e+12 vg	Other

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

Summary

Timeline

Publications

Drugs

Indications