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Sangamo Reports Hemophilia A Gene Therapy Progress at ASH

December 7, 2016
Sangamo Reports Hemophilia A Gene Therapy Progress at ASH

Sangamo BioSciences, Inc., announced the presentation of data on SB-525, the company’s gene therapy program for hemophilia A. The announcement was made at the American Society of Hematology's 58th Annual Meeting December 3-6, 2016, in San Diego, CA.

Sangamo is developing new gene therapies using adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that initiate the production of the deficient factor protein—in this case, factor VIII (FVIII) in those with hemophilia A. The AAVs deliver this genetic material into liver cells, without causing disease or triggering significant immune responses.

According to a Sangamo press release, a single intravenous administration of SB-525 results in the production of “significant levels of FVIII in mice and non-human primates (NHPs) and correction of the bleeding defect in a mouse model of hemophilia A.” In these animals, mean FVIII levels ranged from 5% of normal at the lowest dose to 230% at the highest. These percentages could be therapeutically significant if they can be replicated in humans with hemophilia A.

‘We have developed an improved gene therapy vector for treatment of hemophilia A which we believe is highly competitive, and we remain on track to file an Investigational New Drug (IND) application for our clinical program by the end of 2016,” said Sandy Macrae, MB, ChB, PhD, Sangamo’s president and chief executive officer. 

Source: Sangamo press release dated December 5, 2016