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Graphite Bio Presents Preclinical Data Supporting GPH101 in Sickle Cell Disease at 49th Annual Sickle Cell Disease Association of America National Convention
Data support ability of company’s gene editing platform to precisely and efficiently correct the underlying disease-causing mutation and restore adult
About this update from Lenz Therapeutics, Inc.
[{"type":"text","content":"\nData support ability of company’s gene editing platform to precisely and efficiently correct the underlying disease-causing mutation and restore adult hemoglobin expression with curative potential\n\nData show minimal off-target editing using company’s exclusively licensed high fidelity Cas9 and robust long-term engraftment\n\nCompany on track to enroll first patient in Phase 1/2 CEDAR trial in 2H 2021\n\n SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--\nGraphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, announced today the presentation of preclinical data for GPH101, an investigational therapy designed to directly correct the genetic mutation responsible for sickle cell disease (SCD). Data were presented at the 49th Annual Sickle Cell Disease Association of America (SCDAA) National Convention in a poster presentation.\n\n“These positive preclinical data are foundational to our sickle cell disease program and support the evaluation of GPH101 in our Phase 1/2 CEDAR trial, for which we are on track to enroll our first patient before the end of the year,” said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. “These encouraging data reinforce our belief that GPH101 has the potential to directly correct the underlying disease-causing mutation to decrease production of sickle hemoglobin and restore the expression of normal adult hemoglobin with minimal off-target editing. Gene correction has been viewed as the optimal approach to potentially cure sickle cell disease, and the preclinical data we have generated indicate we can do this precisely and efficiently and at rates that are considered potentially curative based on extensive data from patients who have undergone allogeneic stem cell transplant.”\n\nGraphite Bio presented data establishing the reproducibility of the company’s gene editing platform to generate gene-corrected hematopoietic stem cells (HSCs) for the treatment of SCD. Using Graphite Bio’s gene correction platform, which uses an engineered high fidelity Cas9 to reduce off-target cleavage by 30-fold, the company was able to achieve greater than 60% of gene-corrected beta-globin alleles in vitro with minimal off-target activity. After transplant into mice, long-term en...