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The two presentations 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 presentations will describe the ability to re-create specific HPFH gene variants in the intended target tissue, human primary CD34+ stem cells, and the effect of these gene variants at re-creating the expression of protective fetal hemoglobin. Additional data on progress towards filing an IND or CTA will also be presented.
“We are excited by our progress and we remain focused on our goal of bringing potentially transformative therapies to patients with beta thalassemia and sickle cell disease,” said Dr.
Details of the presentations are as follows:
Title: | CRISPR/Cas9 – Mediated Genome Editing of Human CD34+ Cells Upregulate Fetal Hemoglobin to Clinically Relevant Levels in Single Cell-Derived Erythroid Colonies (Abstract #3623) |
Presenter: | Bibhu Mishra, Ph.D., (Title)), CRISPR Therapeutics |
Date: | Monday, December 5, 2016, 6:00-8:00PM PT - Hall GH (San Diego Convention Center) |
Session: | 112. Thalassemia and Globin Gene Regulation: Poster III |
Title: | Re-Creating Hereditary Persistence of Fetal Hemoglobin (HPFH) to Treat Sickle Cell Disease (SCD) and β-Thalassemia (Abstract #4708) |
Presenter: | Bill (Sven Ante) Lundberg, M.D., M.B.A, Chief Scientific Officer, CRISPR Therapeutics |
Date: | Monday, December 5, 2016, 6:00-8:00PM PT - Hall GH (San Diego Convention Center) |
Session: | 801. Gene Therapy and Transfer: Poster III |
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MEDIA CONTACTS:Jennifer Paganelli W2O Group for CRISPR 347-658-8290 jpaganelli@w2ogroup.com INVESTOR CONTACT:Chris Brinzey Westwicke Partners for CRISPR 339-970-2843 chris.brinzey@westwicke.com