Sangamo BioSciences, Inc.
) jumped more than 17% to $22.96 per share on Mar 6 following
positive data on its HIV candidate SB-728-T. The company
presented encouraging early stage data from the SB-728-T program
at the conference on retroviruses and opportunistic infections
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SB-728-T makes use of the company's zinc finger DNA-binding
protein nucleases (ZFN) genome- editing technology. SB-728-T is
an autologous CD4+ T-cell candidate where the gene for CCR5, a
co-receptor for the dreaded virus to enter, is modified using
ZFN-mediated genome editing to disrupt the CCR5 protein. The
disruption of the CCR5 protein makes the relevant T-cells
resistant to infection by the most common HIV strain.
Data from a phase I /II open-label, dose escalation, multi-center
study (SB-728-1101) revealed that HIV patients treated with
Bristol-Myers Squibb Company
) chemotherapy drug Cytoxan (cyclophosphamide) responded better
to the subsequent SB-728-T infusion. The patients did not take
antiretroviral drugs. The candidate was found to be safe and well
However, the positive results were witnessed for a very small
population. The highest dose of Cytoxan, 1.0 g/m2, prior to
SB-728-T infusion led to the greatest decrease in viral load.
Data revealed that the virus stabilized (but was detectable) in 2
of the 3 patients treated with the dose. Sangamo said that the
study was extended to include six more HIV patients to determine
the optimal dose of the chemotherapy drug. The company intends to
evaluate 12 more HIV patients at the optimal dose.
At the same conference, Sangamo also provided an update on
another ongoing study (SB-728-902, cohort 5) on SB-728-T. Sangamo
said that data from the phase II study revealed that SB-728-T
treatment caused HIV levels to drop significantly in a patient
who was without treatment for 31 weeks.
Sangamo also said that it has developed a new method to use
messenger RNA (mRNA) delivery of the ZFNs used in CCR5 genome
modification of the T-cells. This will be used in the next study
on SB-728-T, expected to commence by Jun 30, 2014. We believe
investor focus will remain on updates on SB-728-T going forward.
We remind investors that Sangamo was also in the news earlier
this year when it inked a deal with
) on the development of therapeutics for hemoglobinopathies
resulting from the abnormal structure or underproduction of
Sangamo currently carries a Zacks Rank #3 (Hold). Better-ranked
) and Biogen. While Alexion carries a Zacks Rank #1 (Strong Buy),
Biogen holds a Zacks Rank #2 (Buy).