Sangamo BioSciences: A Bet on Gene Editing

12/08/2017 5:00 am EST

Focus: HEALTHCARE

John McCamant

Editor, Medical Technology Stock Letter

In a major event for the field of gene editing, Sangamo BioSciences (SGMO) has dosed the first patient in the CHAMPION study of SB 913 in vivo gene editing therapeutic for MPS II, explains John McCamant, biotech expert and editor The Medical Technology Stock Letter.

The strategy of co-opting the albumin gene locus to make a therapeutic protein is compelling given the low cell transfection required for success and bodes well for data expected in 2018 from the company’s multiple gene editing/therapy programs.

According to the company, "For the first time, a patient has received a therapy intended to precisely edit the DNA of cells directly inside the body."

SB 913 is an in vivo genome editing product candidate that utilizes zinc finger nucleases (ZFNs) to insert a copy of the iduronate 2 sulfatase (IDS) gene into a precise location within the albumin gene locus.

The ZFNs, as well as the corrective gene, are delivered using adeno-associated virus serotype 2/6 (AAV2/6) vectors, and the expression of ZFNs are driven by a liver specific promoter.

The CHAMPIONS study is designed to enroll up to 9 adult males with attenuated MPS II (also known as Hunter syndrome). The first patient was treated at the University of California San Francisco (UCSF) Benioff Children’s Hospital Oakland.

Patient screening is also ongoing at additional clinical sites that specialize in MPS II care, including hospitals in Chapel Hill, Chicago, Minneapolis and Philadelphia.


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The current standard of care for MPS II is weekly infusions of enzyme replacement therapy (ERT), and the IDS blood levels quickly return to near undetectable levels within a day of receiving ERT.

The CHAMPIONS study is the first to dose a patient among SGMO’s  three in vivo  gene editing programs, with the other two programs currently screening patients for enrollment in Phase I studies in hemophilia B and MPS I (also known as Hurler Scheie syndrome), respectively.

Multiple catalysts are still approaching in those programs, as well as the Phase I trial in hemophilia A already underway. Lastly, we expect at least one new corporate collaboration with either the tau protein/Alzheimer’s program and/or the cell editing for immuno-oncology/CAR-T.

One of the reasons, in our view, that SGMO’s  valuation lags that of BLUE and ONCE, is that at times both Wall Street and competitors have down played their ZFN platform.

A large part of the negativity is because SGMO’s ZFN patent estate is so solid — others need to develop technologies to get around the exquisiteness of ZFNs. That, we believe, leads to additional reasons for Wall Street to recommend the company leading to further positive share appreciation.

With the new management team now well entrenched, the opportunity to fully maximize SGMO’s  value is upon us. SGMO is a buy under 15 with a target price of 20.

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