Graphite Bio Presents Preclinical Data for Novel Sequencing Method Used to Determine Gene Editing Outcomes at 64th ASH Annual Meeting
Graphite Bio, Inc. (NASDAQ: GRPH) presented preclinical results at the ASH Annual Meeting indicating the efficacy of a single-cell RNA sequencing method to assess gene correction in sickle cell patients treated with nulabeglogene autogedtemcel (nula-cel). This investigational therapy aims to correct the mutation causing sickle cell disease (SCD). The research demonstrated the method's ability to measure and differentiate reticulocyte gene editing outcomes, providing crucial insights for nula-cel's clinical development.
- Preclinical results support the use of single-cell RNA sequencing to measure gene correction outcomes.
- Demonstrated capability of the method to accurately assess reticulocyte gene editing.
- Nula-cel is designed to directly correct the genetic mutation causing SCD, potentially curing the disease.
- None.
Single-cell RNA sequencing of reticulocytes will be used to measure gene correction outcomes in sickle cell patients treated with nulabeglogene autogedtemcel (nula-cel)
“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
Graphite 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.
Based on this knowledge,
The poster is now available on the
Poster Session II: 801. Gene Therapies
Poster #3468: Single-Cell RNA Sequencing of Sickle Cell Reticulocytes to Identify Beta-Globin Genotypes and Associated Gene Expression Differences
Presenting Author:
Date and Time:
Location: Hall D
About nulabeglogene autogedtemcel (nula-cel)
Nula-cel, formerly GPH101, is an investigational gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). A serious, life-threatening inherited blood disorder, SCD affects approximately 100,000 people in
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Statements we make in this press release may include statements that are not historical facts and are considered forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact, including statements regarding the clinical and therapeutic potential of our gene editing platform and our product candidates, including nula-cel, and the value of our novel single-cell RNA sequencing method, may be deemed to be forward-looking statements. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.
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