Date: 2016-07-18
Type of information: Presentation of results at a congress
phase:
Announcement: presentation of results at the ASH Workshop on Genome Editing
Company: Bluebird bio (USA - MA)
Product: megaTAL genome editing platform
Action
mechanism: enzyme/gene therapy
Disease:
Therapeutic area:
Country:
Trial details:
Latest
news: * On July 18, 2016, bluebird bio announced the presentation of pre-clinical data from its megaTAL genome editing platform at the ASH Workshop on Genome Editing, held July 14-15 in Washington, D.C. In an invited presentation entitled “Gene Editing with megaTALs: Advantages, Challenges and Future Prospects,” Jordan Jarjour, Ph.D., director of nuclease research and development, bluebird bio, detailed the anatomy and physiology of megaTALs and discussed how to expand their DNA targeting capacity and refine their specificity. The therapeutic application of genome editing demands technology that is capable of both high on-target activity and exquisite genome-wide specificity. The data reported at the ASH workshop highlight recent progress bluebird has made in expanding the number of megaTAL targetable sites in the genome to permit the fine placement of an editing event within a target gene, in refining the specificity of megaTALs to eliminate undesirable off-target activity and in combining megaTALs targeting different target genes to achieve the knockout of multiple genes simultaneously.
