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Graphite Bio Receives IND Clearance to Initiate Clinical Trial for Next-Generation Gene Editing Therapy GPH101 in Sickle Cell Disease

Photo Credit: Graphite Bio
Dec 14 2020

- IND clearance for Phase 1/2 trial establishes Graphite Bio as the first company advancing potentially curative sickle cell gene editing therapy to restore normal hemoglobin expression

- GPH101 harnesses the power of CRISPR and natural DNA repair mechanisms to precisely insert the correct DNA sequence, with the aim of directly correcting the disease-causing sickle mutation and leading to the production of normal red blood cells

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio, a next-generation gene editing company focused on therapies that harness targeted DNA integration to treat or cure serious diseases, today announced that the U.S. Food & Drug Administration (FDA) has cleared its investigational new drug (IND) application for the experimental gene editing therapy GPH101 to initiate a Phase 1/2 clinical trial in patients with severe sickle cell disease (SCD). 

“The FDA clearance to advance our first investigational therapy, GPH101, into clinical development is a tremendous milestone enabling us to rapidly advance our targeted DNA integration approach into the clinic and bringing Graphite Bio one step closer to making a difference for patients,” said Josh Lehrer, M.Phil., M.D., FACC, chief executive officer at Graphite Bio. “We are eager to initiate enrollment for the CEDAR clinical trial in early 2021, which will be a historic milestone as the first experimental treatment designed to correct the mutation that is the underlying cause of sickle cell disease. With targeted gene integration, GPH101 seeks to restore normal hemoglobin expression, which has been the ultimate goal of sickle cell disease treatment for more than 70 years.”

CEDAR, a Phase 1/2, multi-center, open-label clinical study is designed to evaluate the safety, preliminary efficacy and pharmacodynamics of GPH101 in adult and adolescent patients with severe SCD. An inherited blood disorder affecting 100,000 Americans and millions of people worldwide, SCD is caused by a single mutation in the ß globin gene that leads to damaged and misshapen red blood cells resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span.

GPH101 is Graphite Bio’s lead investigational therapy that will be evaluated as a potentially curative therapy for patients suffering from SCD. The investigational therapy harnesses the power of CRISPR and DNA’s natural homology-directed repair mechanisms to cut out the mutation in the sickle globin gene and paste in the correct natural (wild-type) DNA sequence, with the aim of curing SCD through directly correcting the underlying disease-causing mutation and leading to the production of completely normal red blood cells.

Graphite Bio recently entered into a definitive license agreement for GPH101 with Stanford University, where the investigational therapy’s preclinical development was advanced at the Center for Definitive and Curative Medicine. The agreement includes exclusive rights to develop selected therapies employing efficient genome editing in hematopoietic stem cells by targeted DNA integration in hemoglobinopathies and several other indications. The preclinical development for GPH101 was led by Stanford Medicine with support from the California Institute for Regenerative Medicine (CIRM).

“Today’s announcement represents an important step toward making gene editing by targeted DNA integration a therapeutic reality for patients. Clinical entry for GPH101 represents the culmination of decades of research and scientific investment,” said Matthew Porteus, MD, PhD, co-founder of Graphite Bio. “The scientific platform behind Graphite Bio’s pipeline was born out of a passion for improving the treatment paradigm for people with sickle cell disease, and it is my hope that this platform can one day offer a cure for this and many other devastating diseases.”

About Graphite Bio

Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The company’s targeted DNA integration platform harnesses the natural cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients’ lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit


Christy Curran