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Sangamo BioSciences (SGMO) FDA Clearance Of IND For ZFN-mediated genome editing treatment

February 8, 2016 8:12am

SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I).

The U.S. FDA has cleared SGMO’s IND application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I).

What's the price and will it stay "nice" in a projected ... down day

The SB-318 IND application is now active and enables Sangamo to initiate a P1/2 clinical study (SB-318-1502) designed to assess the safety, tolerability and potential efficacy of SB-318 in adults with varying severities of MPS I.

SB-318 is Sangamo's second in vivo genome editing application cleared by the FDA; the first being for hemophilia B (SB-FIX). Both programs are based on Sangamo's proprietary In Vivo Protein Replacement Platform (IVPRP™), a single treatment strategy designed to produce stable circulating levels of a therapeutic protein from a patient's liver for the lifetime of the individual.

The Bottom Line: The proprietary IVPRP genome editing approach allows SGMO to precisely target and edit the albumin 'safe harbor' locus in the DNA of liver cells, with a single administration, which potentially result in the durable expression of therapeutic enzyme that would be maintained throughout the patient's life.

SB-318 treatment is intended to eliminate the need for enzyme replacement therapy (ERT) which is the current standard of care for the majority of patients with MPS I. ERT for MPS I often requires weekly infusions of a recombinant form of the enzyme alpha-L-iduronidase (IDUA) which is missing, or defective, in patients with the disorder. While the infusions take several hours, circulating levels of IDUA are undetectable within hours of the treatment due to the replacement protein's short half-life.

MPS I, including Hurler syndrome, is tremendously debilitating - and actually life-threatening for affected individuals who struggle with progressive disease even using current therapies.

Conventional AAV gene therapy approaches which are non-integrating and have the potential to "wash out" over time as the patient's liver cells divide and turn over. Ultimately, target population for this approach will be pediatric patients with MPS I, who will benefit most from a one-time, permanent treatment.

SGMO closed DOWN -$0.31 to $5.66 and is UP in the pre-market +0.74. Watch out for strength induced selling as the session wains … still a BUY

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