Accepted abstracts highlight next-generation gene editing programs GPH101 for sickle cell disease and GPH102 for beta-thalassemia

Chief Scientific Officer Dr. Jane Grogan to participate in scientific symposium

Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop therapies with the potential to treat or cure serious diseases, today announced that two abstracts will be presented at the American Society of Gene and Cell Therapy (ASGCT) 25^th Annual Meeting. The abstracts include an oral presentation highlighting the discovery and development of GPH102, the company’s differentiated gene replacement program for beta-thalassemia, as well as an encore of the trial-in-progress poster for the company’s Phase 1/2 CEDAR trial of GPH101, an investigational therapy designed to directly correct the genetic mutation responsible for sickle cell disease. The hybrid meeting will take place virtually and at the Walter E. Washington Convention Center in Washington, D.C., from May 16-19.

“Using our next-generation gene editing platform, we finally have the ability to directly address the genetic lesions that cause serious diseases like sickle cell disease and beta-thalassemia,” said Josh Lehrer, M.D., M.Phil., chief executive officer of Graphite Bio. “We believe our GPH102 program for beta-thalassemia is a natural application of our platform technology. Similar to our GPH101 program for sickle cell disease, our GPH102 program for beta-thalassemia is highly differentiated from other treatments in development in that it has the potential to lead to a definitive cure by normalizing the genetic mutation that causes the disease. We look forward to presenting preclinical data outlining the discovery of GPH102 and supporting its further development.”

The abstracts are now available at annualmeeting.asgct.org. Additional data on GPH102 beyond what is included in the abstract will be presented at the meeting. Details of the presentations follow:

Oral Session: Gene Editing in Blood and Immune Disorders

Abstract 66: Development of a Beta-Globin Gene Replacement Strategy as a Therapeutic Approach for Beta-thalassemia

Presenting Author: Beeke Wienert, Ph.D., associate director, gene engineering, Graphite Bio

Date and Time: Monday, May 16, 2022, 3:45-5:30 p.m. ET

Location: Ballroom A

Poster Session: Gene and Cell Therapy Trials in Progress

Abstract 806: 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 Sickle Cell Disease (SCD)

Presenting Author: John DiPersio, M.D., Ph.D., Washington University School of Medicine

Date and Time: Tuesday, May 17, 2022, 5:30-6:30 p.m. ET

Location: Hall D

In addition to the presentations, Graphite Bio’s Chief Scientific Officer Jane Grogan, Ph.D., will present as part of a scientific symposium on innovations in cell and gene therapies. Details of the presentation follow:

Scientific Symposium: Part 2: Translating Science Into Medicine: Moving from Bench to Startup

Presentation Title: Realizing the Full Promise of Gene Editing to Develop One-time Curative Therapies

Date and Time: Monday, May 16, 2022, 1:30-3:15 p.m. ET

Location: Salon G

About GPH101 for Sickle Cell Disease

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). 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. 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 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 GPH102 for Beta-thalassemia

GPH102 is Graphite Bio’s research program for the treatment of beta-thalassemia, one of the most common autosomal recessive disorders in the world with approximately 68,000 people born with it each year. Beta-thalassemia is a genetic blood disorder characterized by reduced production of beta-globin, a protein that forms oxygen-carrying hemoglobin with alpha-globin. People with the most severe form of beta-thalassemia fail to produce functional beta-globin, which results in severe anemia and transfusion dependency. GPH102 is designed to use Graphite Bio’s gene replacement approach to replace the entire mutated beta-globin gene with a functional gene and restore adult hemoglobin (HbA) expression to levels similar to individuals who do not have disease.

About Graphite Bio

Graphite Bio is a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair 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 UltraHDR™ gene editing platform is designed to precisely correct genetic mutations, replace entire disease-causing genes with functional 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 www.graphitebio.com and following the company on LinkedIn.

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