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Graphite Bio to Host Key Opinion Leader Webinar on Gene Correction for Sickle Cell Disease

Virtual event on Tuesday, December 14, 2021, at 3 p.m. ET to discuss how the company’s differentiated gene correction approach using GPH101 could lead to curative outcomes

Webinar to include an encore of the company’s poster presentation of the Phase 1/2 CEDAR Trial for GPH101 at the upcoming 63rd ASH Annual Meeting and Exposition

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, announced today that it will host a virtual Key Opinion Leader webinar on Tuesday, December 14, 2021, at 3 p.m. ET. The event will highlight Graphite Bio’s differentiated gene correction approach for sickle cell disease (SCD) using GPH101, the company’s lead investigational gene therapy; how GPH101 fits in the current gene therapy landscape; and GPH101’s potential to lead to curative outcomes in SCD.

The event will feature presentations by Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio, along with the following leading professionals in gene editing and SCD:

  • Mitchell J. Weiss, M.D., Ph.D., St. Jude Children’s Research Hospital, will provide an overview of the SCD gene therapy landscape.
  • Matthew H. Porteus, M.D., Ph.D., Stanford University School of Medicine and academic founder of Graphite Bio, will discuss the foundational research for the company's differentiated gene correction approach for SCD.
  • Julie Kanter, M.D., University of Alabama at Birmingham, will provide an encore of the company’s poster presentation of the Phase 1/2 CEDAR trial for GPH101 at the 63rd ASH Annual Meeting and Exposition. The original presentation will take place on Saturday, December 11, 2021, from 5:30-7:30 p.m. ET.
  • John F. DiPersio, M.D., Ph.D., Washington University School of Medicine, will review the criteria for long-term cure in SCD.

The panelists will be available to answer questions following the formal presentations.

The event will be webcast live from Graphite Bio’s website at in the Investors section. A replay of the webinar will be archived and available following the event.

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 to red blood cells that are responsible for delivery oxygen to tissues and organs throughout the body to become misshapen, resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life expectancy of approximately 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 utilize 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 GPH101 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.

Learn more about Graphite Bio by visiting and following the company on LinkedIn.


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