|CRISPR Therapeutics Announces the Presentation of Data on its Lead Program at the Upcoming 22nd European Hematology Association Annual Congress|
This abstract, selected by the Scientific Program Committee as one of the five best abstracts, will highlight recent work in re-creating the natural condition of hereditary persistence of fetal hemoglobin (HPFH) that is protective in sickle cell disease and beta thalassemia. The presentation will describe the ability to re-create specific HPFH gene variants in the intended target tissue, human primary CD34+ stem cells, the effect of these gene variants at re-creating the expression of protective fetal hemoglobin and progress in developing a product ready for clinical testing.
“We are pleased to have been selected as one of the five best abstracts at the leading global conference in the field of hematology. We remain on track to file a clinical trial application by the end of 2017 and the initiation of clinical trials in
Details of the presentation are as follows:
A copy of the EHA abstracts were made available
CRISPR Therapeutics is a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR / Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The company's multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Additionally, CRISPR Therapeutics has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in diseases with high unmet need. The foundational CRISPR / Cas9 patent estate for human therapeutic use was licensed from the company's scientific founder Emmanuelle Charpentier, Ph.D. CRISPR Therapeutics AG is headquartered in Basel,