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Latest announcements and gatherings
The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease.
A Phase 1/2 clinical trial (NCT03745287) is recruiting patients.
CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells.
Fetal hemoglobin is a form of hemoglobin that naturally exists when one is born and later is replaced by the adult form of hemoglobin.
For the treatment, doctors collect a patient’s own hematopoietic stem cells (cells from the bone marrow) and modify them in the laboratory so they can produce high levels of fetal hemoglobin.
videos & visualsJennifer Doudna on ethics of gene editinghttps://www.youtube.com/watch?v=8Ijr1ccY...
news & eventsMaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
news & eventsStem cell gene-editing method may be breakthrough for sickle cell researchResearchers have cured mice with a genet...
news & eventsCRISPR deployed to combat sickle-cell anaemiaA mutation in a single DNA letter causes...
education & researchThe Association Between Timely Opioid Administration and Hospitalization in Children With Sickle Cell Disease Presen...Objective: To evaluate the association ...
news & eventsBioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sick...Bioverativ Inc., a Sanofi company dedi...
news & eventsBioverativ and Bicycle Therapeutics Enter into Strategic Research Collaboration to Develop Therapies for Hemophilia ...Bioverativ Inc. (NASDAQ: BIVV), a global...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.