Breaking News

Sangamo BioSciences, CIRM In Strategic Pact

May 24, 2013

To develop ZFP therapeutic for blood disease

Sangamo BioSciences has received a $6.4 million Strategic Partnership Award from California Institute for Regenerative Medicine (CIRM) to use its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs) to develop a potential ZFP Therapeutic for beta-thalassemia.
 
The four-year grant provides matching funds for preclinical work to support an IND application and a Phase I trial in transfusion-dependent beta-thalassemia, a genetic blood disease caused by mutations in the beta-globin gene.
 
"CIRM support for this program is yet another major validation of our ZFP Therapeutics platform," said Edward Lanphier, Sangamo's president and chief executive officer. "The award will assist the development of our stem cell-based ZFP Therapeutic for the potential cure of thalassemia. Importantly, this same approach can be directly applied to related hemoglobinopathies such as sickle cell disease. We look forward to working with a team of world-renowned experts in this field, including the team at Children's Hospital & Research Center Oakland and our colleagues at CIRM, to bring this treatment through IND application and into a Phase I trial."

Related Contract Manufacturing:

blog comments powered by Disqus
  • Solid Dispersions

    Solid Dispersions

    Robert Harris, Juniper Pharma Services||April 5, 2016
    A universal formulation strategy for poorly soluble drugs?

  • Fit-For-Purpose Assay Development in Bioanalysis

    Fit-For-Purpose Assay Development in Bioanalysis

    Franklin Spriggs, Ligand Binding Assay Group Leader; Ashley Brant, Program Manager, AIT Bioscience||January 28, 2016
    The success of bioanalytical studies relies on the selection of the most suitable analytical method but the timeline of method development and types of analyses involved vary greatly.

  • CMC Biologics: Growing Through Innovation

    Tim Wright, Editor||January 28, 2016
    The new chief exec talks about industry trends and growth plans.