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Graphite Bio Presents Preclinical Data for Novel Sequencing Method Used to Determine Gene Editing Outcomes at 64th ASH Annual Meeting
Single-cell RNA sequencing of reticulocytes will be used to measure gene correction outcomes in sickle cell patients treated with nulabeglogene autogedtemcel
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[{"type":"text","content":"\nSingle-cell RNA sequencing of reticulocytes will be used to measure gene correction outcomes in sickle cell patients treated with nulabeglogene autogedtemcel (nula-cel)\n\n SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--\nGraphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop therapies with the potential to treat or cure serious diseases, today presented preclinical results supporting the use of a single-cell RNA sequencing method to assess gene correction outcomes in patients treated with nulabeglogene autogedtemcel (nula-cel), an investigational gene-edited therapy for sickle cell disease (SCD). The findings are being presented in a poster session at the 64th American Society of Hematology (ASH) Annual Meeting & Exposition taking place virtually and at the Ernest N. Morial Convention Center in New Orleans.\n\n“Our goal is to cure sickle cell disease by directly correcting the underlying disease-causing genetic mutation in order to simultaneously reduce sickle hemoglobin production and restore healthy adult hemoglobin expression, thereby potentially alleviating all complications associated with the disease,” said Josh Lehrer, M.D., M. Phil., chief executive officer of Graphite Bio. “The novel single-cell RNA sequencing method that we developed will help us determine initial gene editing outcomes in patients treated with nula-cel, providing important preliminary information about the potential efficacy of the investigational therapy.”\n\nGraphite Bio’s gene correction approach for SCD involves editing hematopoietic stem cells found in the bone marrow that develop into various types of blood cells such as red blood cells. Since red blood cells lose their nucleus and genomic DNA during maturation, tracking gene editing outcomes in mature red blood cells via nucleic acid sequencing is not possible. However, immature red blood cells called reticulocytes retain RNA that can be sequenced in order to assess gene correction levels.\n\nBased on this knowledge, Graphite Bio scientists sought to develop a single-cell RNA sequencing method that could measure gene editing outcomes in reticulocytes. To establish proof-of-concept and evaluate the accuracy of the method, researchers measured the genetic makeup of reticulocytes from healt...