The phase I/II study to evaluate the effect of SB-913, an investigational in vivo genome editing therapy, in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Phase of Trial: Phase I/II
Latest Information Update: 22 Nov 2017
At a glance
- Drugs SB 913 (Primary)
- Indications Mucopolysaccharidosis II
- Focus Adverse reactions; Pharmacokinetics; Therapeutic Use
- Acronyms CHAMPIONS study
- 22 Nov 2017 New trial record
- 15 Nov 2017 According to a Sangamo Therapeutics media release, Paul Harmatz, M.D., a pediatric gastroenterologist is a principal investigator of the study.
- 15 Nov 2017 According to a Sangamo Therapeutics media release, first patients has been dosed in this trial.