2018-000192-33: A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in patients with Mucopolysaccharidosis II (MPS II) |
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| Not yet recruiting | 1/2 | 32 | Europe | rAAV2/6 Left ZFN Vector, rAAV2/6 Right ZFN Vector, rAAV 2/6 hIDS DONOR, SB-47171, SB-47898, hIDS DONOR, Solution for infusion | Sangamo Therapeutics, Inc., Sangamo Therapeutics, Inc. | Mucopolysaccharidosis type II (MPS II), Hunter Syndrome, is a rare, inherited disease caused by a deficiency in an enzyme called iduronate-2-sulfatase. This causes glycosaminoglycans (GAGs) to build up in the body and cause damage., Diseases [C] - Nutritional and Metabolic Diseases [C18] | | | | |
