SB-318 is Sangamo's second in vivo genome editing application cleared by the
"MPS I, including Hurler syndrome, is tremendously debilitating - and actually life-threatening for affected individuals who struggle with progressive disease even using current therapies," said
"Our proprietary IVPRP genome editing approach allows us to precisely target and edit the albumin 'safe harbor' locus in the DNA of liver cells, with a single administration, which we expect to result in the durable expression of therapeutic enzyme that would be maintained throughout the patient's life," said
"Genome editing has the potential to change the way medicine is practiced, and we have demonstrated that our zinc finger nuclease technology leads the field in the development of therapeutics for both in vivo and ex vivo applications," said
Sangamo remains on track to file additional IND applications for programs employing the IVPRP approach, including MPS II (Hunter syndrome) in the first half of 2016, and hemophilia A, Gaucher disease and Fabry disease in the second half of 2016.
SB-318-1502 is a Phase 1/2 open-label, dose-escalation study in subjects over eighteen years of age with varying severities of MPS I, including Scheie, Hurler-Scheie and Hurler syndrome. The study will begin enrolling up to nine subjects in mid-2016, with the possibility of expanding to 12 subjects, to evaluate the safety, tolerability and efficacy of a single administration of SB-318. The principal investigator,
About Sangamo's IVPRP™
The IVPRP approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic "safe-harbor site," that can be edited with zinc finger nucleases (ZFNs) to accept and express any therapeutic gene. The platform is designed to enable the patient's liver to permanently produce circulating, therapeutic levels of a corrective protein product, such as Factor VIII or IX to treat hemophilia, or replacement enzymes to treat lysosomal storage disorders. The ability to permanently integrate the therapeutic gene in a highly specific targeted fashion significantly differentiates Sangamo's IVPRP approach from conventional AAV gene therapy approaches, which are non-integrating and may "wash out" of the liver as cells divide and turn over. Ultimately, the target population for IVPRP programs will be pediatric patients for whom it is critical to be able to produce stable levels of therapeutic protein for the lifetime of the patient. With such a large capacity for protein production (approximately 15g/day of albumin), targeting and co-opting only a very small percentage of the albumin gene's capacity is sufficient to produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production. The first two IVPRP clinical programs, for hemophilia B and MPS I, both received unanimous approval from the
About MPS I / Hurler Syndrome
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder, caused by mutations in the gene encoding the alpha-L-iduronidase (IDUA) enzyme, resulting in a deficiency of IDUA which is required for the degradation of the glycosaminoglycans (GAGs), dermatan sulfate and heparin sulfate. The inability to degrade GAGs leads to their accumulation within the lysosomes throughout the body and individuals with this mutation experience multi-organ dysfunction and damage. Depending on the severity of the mutations and degree of residual enzyme activity, affected individuals may develop organomegaly, joint stiffness, skeletal deformities, corneal clouding, hearing loss and mental retardation. Three forms of MPS I, in order of increasing severity, include Scheie, Hurler-Scheie and Hurler syndromes. According to the
ZFP Therapeutic® is a registered trademark of
This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFNs and therapeutic applications of Sangamo's ZFP technology platform, the potential of Sangamo's ZFP technology to treat hemophilia B, hemophilia A and lysosomal storage disorders, including MPS I, MPS II, Gaucher disease and Fabry disease, the expected timing of trial enrollment for SB-318-1502 and filing of IND applications for hemophilia A, MPS II Gaucher disease and Fabry disease, the impact of the SB-318-1502 clinical trial on the field of genetic medicine, the potential benefits of SB-318 as treatment for pediatric patients, and the safety and efficacy of the approach of using ZFN-mediated genome editing. Actual results may differ materially from these
forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the safety, tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's public filings with the
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