Sangamo Biosciences, Inc. (NASDAQ:SGMO) recently announced that it presented its manufacturing & preclinical data on Hemophilia, at the 2016 American Society of Hematology Meeting. The preclinical data aims at SB-525, which is a gene therapy for hemophilia A. The 58TH Annual Meeting of ASH was organized from December 3-6, 2016 at San Diego, California.
Sangamo Biosciences to file Investigational New Drug
The company is confident that its gene therapy vector will prove to be highly fruitful for hemophilia A treatment. Also, the company said, it will prove to be highly competitive in the market with lucrative gains. Sangamo Biosciences has decided to file the Investigational New Drug or IND soon. The company said that the new programme depicts its capabilities that do not remain constricted to ZFP platform.
With this new manufacturing & preclinical data on Hemophilia, the company has expanded the technical and R&D sector as well. The company has also swelled its clinical development capabilities and has made certain changes to senior management.
There are four other clinical programmes of the company that are lined up or ongoing. So as to understand and comprehend SB-525, the company said that it is also looking forward to the clinical trial that will kick off next year.
Useful results on non-human primates
The company explained that a single-time use of SB-525 can lead to vital changes in level of hFVII in mice as well as non-human primates. It has also resulted in improved bleeding defect for hemophilia A in the mouse model. This has also shown changes in AAV yields through the process of vector manufacturing. The company said that the most important thing was reproducible and a strong curve of dose response. This, Sangamo Biosciences said, has so far been the most compelling dose response recorded in non-human primates. Further data of other clinical trials shall be presented in the times to come, but this representation was an important one.