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Graphite Bio (NASDAQ: GRPH) announced that information regarding its Phase 1/2 CEDAR trial of GPH101 for sickle cell disease (SCD) will be presented at the 63rd American Society of Hematology Annual Meeting from December 11-14. GPH101 aims to correct the genetic mutation causing SCD, potentially curing the disease. The trial assesses safety, engraftment success, and gene correction rates. SCD affects around 100,000 people in the U.S., highlighting the urgent need for effective therapies.
Positive
GPH101 aims to directly correct the genetic mutation causing sickle cell disease, potentially offering a cure.
The CEDAR trial is a Phase 1/2 clinical study, providing early insights into therapy effectiveness and safety.
Presentation at a prominent medical conference increases visibility and awareness of GPH101.
Negative
None.
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--
Graphite 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, today announced that information about the company’s Phase 1/2 CEDAR trial of GPH101, an investigational therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD), will be presented during a poster session at the 63rdAmerican Society of Hematology (ASH) Annual Meeting & Exposition. The meeting will take place virtually and at the Georgia World Congress Center in Atlanta from December 11-14.
The ASH abstract is now available at www.hematology.org. Details of the Graphite Bio poster presentation are as follows:
Poster Session: 801. Gene Therapies: Poster I
Poster #1864: CEDAR Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD
Presenting Author:Julie Kanter, M.D., University of Alabama at Birmingham Date and Time:Saturday, Dec. 11, 9 a.m.-7:30 p.m. ET
About Sickle Cell Disease (SCD)
SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. SCD is caused by a single mutation in the beta-globin gene that leads red blood cells to become misshapen, resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life expectancy of approximately 20-30 years. Despite advancements in treatment and care, progressive organ damage continues to cause early mortality and severe morbidity, highlighting the need for curative therapies.
About GPH101
GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore normal adult hemoglobin (HbA) expression, thereby potentially curing SCD.
Graphite Bio is evaluating GP101 in the CEDAR trial, an open-label, multi-center Phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing company harnessing high efficiency targeted gene integration to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases. Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. Graphite Bio’s platform allows it to precisely correct mutations, replace entire disease-causing genes with normal genes or insert new genes into predetermined, safe locations. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.
What is the purpose of the CEDAR trial conducted by Graphite Bio (GRPH)?
The CEDAR trial aims to evaluate the safety, engraftment success, gene correction rates, and overall effectiveness of GPH101 in treating sickle cell disease.
When will the results of the GPH101 trial be presented?
The results of the GPH101 trial will be presented during a poster session at the American Society of Hematology Annual Meeting from December 11-14.
How many people are affected by sickle cell disease in the U.S.?
Approximately 100,000 people in the United States are affected by sickle cell disease.
What innovative approach does GPH101 use to treat sickle cell disease?
GPH101 employs next-generation gene editing to directly correct the genetic mutation in the beta-globin gene, potentially restoring normal hemoglobin production.
