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The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
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CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.
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Case: Langston is a 12-year-old with sickle cell disease whom you have followed in your practice since infancy.
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CHOP researchers have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells.
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A group of researchers that includes CHOP presented new data on CRISPR-edited gene therapy for blood disorders at an international meeting.
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CHOP researchers have identified two transcription factors that are involved in silencing fetal hemoglobin in adult red blood cells.
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A collaborative team of researchers, including Dr. Stephan Grupp, recently presented preliminary data showing that a CRISPR-based gene-editing therapy for inherited blood disorders is safe and effective.
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Kwaku Ohene-Frempong, MD was named a 2021 Honorific Award Recipient by the American Society of Hematology.
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CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.
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The US Food and Drug Administration has approved two new breakthrough therapies for patients with sickle cell disease (SCD), and we believe both may enhance the treatment of our patients here at CHOP.