Review of Current Gene Therapy Market - Snngamo Mention (SGMO)

Pharmavoice.com has a new online review of the gene therapy market.
Here is the relevant Sangamo excerpt:

Biogen has a collaboration with Sangamo BioSciences in the area of zinc finger nucleases related to hemoglobinopathies, targeting sickle cell disease and beta-thalassemia. The beta-thalassemia program was initiated with a BCL11a knockout strategy, and the sickle cell program already employs the BCL11A enhancer approach.

Both beta-thalassemia and sickle cell manifest several months after birth, when patients’ cells switch from producing functional fetal globin to a mutant form of adult beta-globin, which causes their condition. The development program uses ZFN-mediated genome editing of a patient’s own hematopoietic stem and progenitor cells (HSPCs) to increase production of fetal globin in cells that will ultimately become red blood cells.

Sangamo and Biogen have developed two related but distinct ZFN-mediated genome editing approaches to disrupt critical aspects of the regulatory pathway that, in early infancy, lead to the switch in production from fetal to adult globin.

Initially, the companies developed a strategy for beta-thalassemia that specifically knocked out the gene encoding for the BCL11A transcription factor, a critical regulator of the switch from fetal to adult globin production.

A second approach was initiated for the sickle cell program, which involved the disruption of the more recently described erythroid-specific enhancer of BCL11A expression, a regulatory DNA sequence in the genome that is essential for expression of BCL11A but that is functional exclusively in cells destined to become RBCs.

Dr. Danos says clinical trials for both programs are expected to begin in 2016.

Read the entire article here:

http://www.pharmavoice.com/article/2015-09-gene-therapy/