Sangamo Therapeutics, Inc. (SGMO) today announced treatment of the first patient in the Phase 1/2 clinical trial ("the CHAMPIONS study") evaluating SB-913, an investigational in vivo genome editing therapy for people with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
A man may soon be forever free of the previously incurable disease he was born with 44 years ago. On Monday, in a medical first, Brian Madeux received an experimental in-body gene-editing treatment intended to cure him of Mucopolysaccharidoses II, known as MPS II or Hunter syndrome, a rare disorder that causes progressive damage to the body's cells.
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